Category: regulatory

Biogen gets much-needed pipeline lift with speedy ALS drug review

amyotrophic lateral sclerosis, antisense oligonucleotides, Biogen, Market Access, neurology, News, regulatory, tofersen / July 26, 2022 July 26, 2022

Beleaguered Biogen has been handed some encouraging news from the FDA, after the regulator agreed to complete its review of amyotrophic lateral sclerosis (ALS) therapy tofersen within six months. The priority review of the Ionis-partnered SOD-1-targeted antisense drug marks the latest stage in the resurrection of the programme, which was all-but written off last October …

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Pfizer, Flynn fined £70m in resurrected epilepsy drug pricing probe

competition law, drug pricing, epilepsy, Financial, Flynn Pharma, News, Pfizer, regulatory, Sales and Marketing, UK / July 22, 2022 July 22, 2022

Drugmakers Pfizer and Flynn Pharma have been fined £70 million ($84 million) by the UK Competition and Markets Authority (CMA) for overcharging the NHS for a widely-used epilepsy drug. The CMA delivered a preliminary judgment in the case last year which concluded that Pfizer and Flynn abused a dominant position in phenytoin sodium capsules, causing …

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EMA pilot will see if clinical trial data should be digested ‘raw’

clinical research, Clinical Trials, data, digital, EMA, News, R&D, regulatory / July 14, 2022 July 14, 2022

A pilot study has been launched by the EMA to see whether it is helpful for regulators to look at the ‘raw data’ from clinical trials submitted in support of new marketing application along with the usual structured analyses from sponsors. The pilot will focus on individual patient data from clinical studies in electronic structured …

EMA pilot will see if clinical trial data should be digested ‘raw’ Read More »

HRA Pharma bids to bring first OTC contraceptive pill to US

Contraception, HRA Pharma, Market Access, News, OTC, patients, Perrigo, regulatory, womens health / July 12, 2022 July 12, 2022

Perrigo’s HRA Pharma subsidiary has become the first drugmaker in the US to seek approval for an over-the-counter contraceptive pill. The move comes as access to birth control becomes a pivotal issue following the landmark ruling by the Supreme Court last month to strip US women of their constitutional protection for the right to abortion, …

HRA Pharma bids to bring first OTC contraceptive pill to US Read More »

Eisai/Biogen could get FDA verdict on new Alzheimer’s drug by 6 Jan

Aduhelm, alzheimer's disease, Biogen, Eisai, FDA, lecanemab, Market Access, neurology, News, regulatory / July 6, 2022 July 6, 2022

The FDA has started an accelerated review of Eisai and Biogen’s a new amyloid-targeting antibody lecanemab for Alzheimer’s disease, with a 6 January deadline that keep them ahead of closest rival Eli Lilly. The US regulator has been reviewing lecanemab as a treatment for patients with early-stage Alzheimer’s and amyloid plaques in the brain under …

Eisai/Biogen could get FDA verdict on new Alzheimer’s drug by 6 Jan Read More »

FDA rejection of Spero’s tebipenem ‘raises resistance threat’

antibiotic, Antimicrobial Resistance, FDA, Market Access, News, regulatory, Spero Therapeutics, tebipenem / June 30, 2022 June 30, 2022

The FDA’s decision not to approve Spero Therapeutics’ oral antibiotic tebipenem Hbr for adults with complicated urinary tract infections (cUTI) further increases the threat of antibiotic resistance, according to a GlobalData analyst. The FDA delivered a blow to Spero’s hopes of bringing the carbapenem to market in a complete response letter (CRL) delivered this week, …

FDA rejection of Spero’s tebipenem ‘raises resistance threat’ Read More »

US panel backs Omicron-targeting vaccines for booster campaigns

BioNTech, coronavirus, COVID-19, Covid-19 Vaccine, FDA, GlaxoSmithKline, Market Access, Moderna, News, Novavax, Omicron, Pfizer, R&D, regulatory, variant / June 29, 2022 June 29, 2022

Future booster campaigns against COVID-19 should include vaccines with a component targeting the Omicron variant of SARS-CoV-2, according to the FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC). After hours of deliberation, the panellists voted by 19 to two in favour of using redesigned booster shots, rather than simply using the currently authorised vaccines …

US panel backs Omicron-targeting vaccines for booster campaigns Read More »

Sarepta shares yo-yo as FDA places hold on Duchenne trial

Duchenne muscular dystrophy, Exondys 51, FDA, News, R&D, rare disease, regulatory, safety, Sarepta, vesleteplirsen / June 24, 2022 June 24, 2022

Shares in Sarepta came under pressure after the biotech said it had temporarily halted a clinical trial of its new-generation Duchenne muscular dystrophy candidate, after a patient had a serious safety incident. The FDA has ordered a clinical hold on Sarepta’s SRP-5051 (vesleteplirsen) drug candidate after one patient in the study developed very low levels …

Sarepta shares yo-yo as FDA places hold on Duchenne trial Read More »

FDA advisors back COVID jabs for very young children

access, BioNTech, coronavirus, COVID-19, Covid-19 Vaccine, Market Access, Moderna, News, paediatric, patients, Pfizer, regulatory / June 16, 2022 June 16, 2022

The FDA’s vaccines advisors have voted in favour of extending the use of mRNA-based COVID-19 vaccine from Moderna and Pfizer/BioNTech to include children from the ages of six months. The Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted 21 to zero in favour of both vaccines in this age group, in light of rising …

FDA advisors back COVID jabs for very young children Read More »

Mallinckrodt tries again with kidney drug terlipressin

kidney disease, Mallinckrodt, News, regulatory, Sales and Marketing, terlipressin / June 15, 2022 June 15, 2022

Manufacturing problems wreaked havoc with Mallinckrodt’s first attempt to secure FDA approval of its treatment candidate terlipressin for hepatorenal syndrome (HRS), but the drugmaker is having another attempt. The Dublin, Ireland-headquartered company said the resubmission will allow more time for the FDA to inspect a packaging and labelling manufacturing facility for the drug, which as …

Mallinckrodt tries again with kidney drug terlipressin Read More »

Alliance eyes European ‘single market’ for digital therapeutics

digital, digital health, Digital Therapeutic Alliance, Digital Therapeutics, harmonisation, Healthware, News, regulatory, reimbursement / June 14, 2022 June 14, 2022

There’s a growing body of evidence that digital therapeutics (DTx) can complement other therapies for a wide range of health conditions, reducing costs and improving patient engagement, but the sector runs the risk of being held back by a lack of clinical, regulatory and reimbursement standards. A new partnership between the non-profit Digital Therapeutics Alliance …

Alliance eyes European ‘single market’ for digital therapeutics Read More »

Second unanimous FDA adcomm vote boosts bluebird bio

beta thalassaemia, bluebird bio, FDA, gene therapy, Market Access, News, rare disease, regulatory / June 12, 2022 June 12, 2022

bluebird bio started last week on tenterhooks, as FDA advisors cast their eyes over data for two gene therapy candidates that are key to its future as a commercially viable concern. By the end of the week, it was two for two, with both recommended for approval by a unanimous vote. Thursday saw the Cellular, …

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FDA panel backs bluebird’s CALD gene therapy, despite safety worries

bluebird bio, CALD, FDA, gene therapy, Market Access, News, rare disease, regulatory / June 10, 2022 June 10, 2022

The FDA may have safety concerns abut bluebird bio’s gene therapy for rare, fatal disease cerebral adrenoleukodystrophy (CALD), but its advisors believe its benefits far outweigh the risks. Members of the agency’s Cellular, Tissue and Gene Therapies advisory committee voted 15 to 0 in favour of approval of elivaldogene autotemcel – also known as eli-cel …

FDA panel backs bluebird’s CALD gene therapy, despite safety worries Read More »

MHRA gives Alnylam ‘innovation passport’ for hypertension drug zilebesiran

Alnylam, HEOR, Hypertension, innovation passport, Market Access, MHRA, News, NHS, regulatory, UK, zilebesiran / June 8, 2022 June 8, 2022

The UK medicines regulator has awarded Alnylam’s RNAi-based therapy zilebesiran for hypertension an ‘innovation passport‘, a designation designed to speed up NHS access to promising new medicines. The passport means that LentiGlobin will be reviewed by the Medicines and Healthcare products Regulatory Agency (MHRA) via the new innovative licensing and access pathway (ILAP) introduced in …

MHRA gives Alnylam ‘innovation passport’ for hypertension drug zilebesiran Read More »

FDA grants speedy review to Dupixent rare skin disease

dermatology, Dupixent, News, prurigo nodularis, rare disease, Regeneron, regulatory, Sales and Marketing, Sanofi / May 31, 2022 May 31, 2022

Sanofi and Regeneron should hear from the FDA in the autumn whether it will approve their blockbuster immunology drug Dupixent as a therapy for rare skin disease prurigo nodularis (PN). The US regulator has granted Dupixent (dupilumab) a six-month priority review in PN and is due to deliver a verdict by 30 September, offering patients …

FDA grants speedy review to Dupixent rare skin disease Read More »

How to keep up with changing payer requirements

appropriate, claim denials, Claim Scrubber, claims, Claims & Contract Management, claims and contract management, Contract Manager, contract manager and contract analysis, denied claims, Featured, No Surprises Act, payer alerts, Payer Compliance, payer requirements, Payers, regulatory, reimbursements / May 25, 2022 May 25, 2022

The payer policy rollercoaster has taken a few twists and turns recently, leaving healthcare organizations out of the loop if they try to keep pace with payer requirements using manual systems alone. Keeping track of changing payer requirements has long been a major challenge for providers, but several shifts in the reimbursement landscape have prompted …

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Nymox slumps as FDA rejects filing for prostate drug

benign prostatic hyperplasia, fexapotide, Market Access, News, Nymox Pharma, regulatory / May 24, 2022 May 24, 2022

Shares in US biotech Nymox Pharma have plunged after the company announced the FDA had refused to accept its marketing application for its drug candidate for benign prostatic hyperplasia (BPH), a common condition affecting older men. The regulator has asked for additional longer-term safety data on the drug, called fexapotide triflutate, which was submitted for …

Nymox slumps as FDA rejects filing for prostate drug Read More »

Digital health body publishes best practice guide for DTx developers

digital, digital health, Digital Therapeutics, Digital Therapeutics Alliance, harmonisation, News, regulatory / May 18, 2022 May 18, 2022

Companies trying to bring digital therapeutic (DTx) products through development and onto the market have been navigating in uncharted waters for years. Now, trade organisation the Digital Therapeutics Alliance has provided a guide to help them during the journey. The DTx Value Assessment & Integration Guide lays out the DTA’s thinking on how developers can …

Digital health body publishes best practice guide for DTx developers Read More »

FDA rejects UCB’s psoriasis therapy bimekizumab

bimekizumab, FDA, Market Access, News, psoriasis, regulatory, UCB / May 13, 2022 May 13, 2022

The FDA has delivered a blow to UCB’s late-stage pipeline, after saying it is unable to approve its latecomer IL-17 inhibitor bimekizumab for moderate-to-severe plaque psoriasis. The Belgian biotech said today it had received a complete response letter from the US regulator “cannot approve the application in its current form” because observations in FDA inspections …

FDA rejects UCB’s psoriasis therapy bimekizumab Read More »

Exit Aduhelm, enter lecanemab. Biogen and Eisai have another go

Aduhelm, alzheimer's disease, amyloid, Biogen, Eisai, lecanemab, Market Access, News, regulatory / May 10, 2022 May 10, 2022

Here we go again. With Aduhelm largely sidelined, Biogen and Eisai are hoping for a better fortunes with their next Alzheimer’s disease therapy lecanemab, which is now fully filed with the FDA. The two partners have also requested a priority review for the Biologics License Application (BLA) for lecanemab, which if granted by the FDA …

Exit Aduhelm, enter lecanemab. Biogen and Eisai have another go Read More »

FDA says no to two more China-developed cancer drugs

China, Coherus, FDA, Hutchmed, Immuno-Oncology, Market Access, News, Oncology, regulatory / May 2, 2022 May 2, 2022

The FDA has declined to approve two more cancer therapies developed by drugmakers based in China, in what may be further evidence of a tough line taken by the regulator for drugs tested outside the US. The agency has sent a complete response letter (CRL) to Hutchmed indicating that its application to market multikinase inhibitor …

FDA says no to two more China-developed cancer drugs Read More »

Ampio slumps as FDA delivers knee osteoarthritis shock

Ampio Pharmaceuticals, Ampion, Market Access, News, Osteoarthritis, R&D, regulatory / April 22, 2022 April 22, 2022

Ampio Pharma’s candidate therapy for knee osteoarthritis has been knocked back by the FDA, which will likely now require a new clinical trial of the drug before it will consider a review. Shares in the US biotech have fallen sharply after it revealed that the regulator did not accept changes to a phase 3 trial …

Ampio slumps as FDA delivers knee osteoarthritis shock Read More »

Teva’s long-acting schizophrenia drug rejected by FDA

Market Access, MedinCell, News, psychiatry, regulatory, Schizophrenia, Teva / April 20, 2022 April 20, 2022

Teva’s long haul to a turnaround of its business has been dealt a blow by the FDA, which has just declined to approve the company’s long-acting formulation of schizophrenia drug risperidone. The US regulator issued a complete response letter (CRL) for the MedinCell-partnered drug, and the companies haven’t been forthcoming on the reasons behind the …

Teva’s long-acting schizophrenia drug rejected by FDA Read More »

How to prepare for CMS’s Appropriate Use Criteria Program

appropriate use criteria, claim denials, claims, Claims & Contract Management, claims denials, claims management, claims management system, claims scrubber, Featured, medical necessity, regulatory, regulatory updates / April 11, 2022 April 11, 2022

The Appropriate Use Criteria (AUC) program is due to come into full effect in January 2023 at the earliest. The program was established by the Centers for Medicare and Medicaid Services (CMS) to help providers order the most appropriate diagnostic imaging services. Failure to comply may result in claims denials. Any provider that offers these …

How to prepare for CMS’s Appropriate Use Criteria Program Read More »

FDA says no to AZ’s Fasenra for nasal polyp indication

AstraZeneca, Dupixent, Fasenra, GlaxoSmithKline, Nasal Polyps, News, Nucala, Regeneron, regulatory, Sanofi / March 14, 2022 March 14, 2022

AstraZeneca has failed in its bid to get US approval for its IL-5 inhibitor Fasenra as a treatment for chronic rhinosinusitis in people with nasal polyps, after the FDA rejected its marketing application. The US regulator has asked for additional clinical data, according to AZ, which said it remains committed to brining the drug to …

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Chasing rivals, Bayer files Nubeqa for new prostate cancer use

Bayer, News, Nubeqa, Oncology, Orion, prostate cancer, regulatory / March 10, 2022 March 10, 2022

Bayer has filed for approval to extend the use of its prostate cancer therapy Nubeqa as it tries to achieve labelling parity with rival drugs from Pfizer/Astellas and Johnson & Jonson, and fulfil its aim of €3 billion in peak sales for the drug. Nubeqa is already making headway in the market as a treatment …

Chasing rivals, Bayer files Nubeqa for new prostate cancer use Read More »

FDA turns down Amryt’s epidermolysis bullosa drug

Amryt Pharma, dermatology, epidermolysis bullosa, inherited disease, Market Access, News, rare disease, regulatory / February 28, 2022 February 28, 2022

Amryt Pharma has been handed a major blow by the FDA, after the US regulator rejected its marketing application for Oleogel-S10, its drug for rare and debilitating skin disease epidermolysis bullosa (EB). Shares in the Ireland-based rare disease specialist fell sharply after news of the complete response letter (CRL) from the FDA emerged, with the …

FDA turns down Amryt’s epidermolysis bullosa drug Read More »

FDA knocks back Mallinckrodt’s kidney drug terlipressin

kidney disease, liver disease, Mallinckrodt, Market Access, News, regulatory, terlipressin / February 23, 2022 February 23, 2022

Manufacturing problems seem to have scuppered Mallinckrodt’s hopes of bringing the first approved drug to market in the US for hepatorenal syndrome (HRS), a life-threatening condition that causes scarring and fluid build-up in the kidney, at least for now. The FDA has issued a complete response letter to Mallinckrodt for terlipressin, on the grounds that …

FDA knocks back Mallinckrodt’s kidney drug terlipressin Read More »

AbbVie files Vraylar as add-on therapy for depression in US

AbbVie, depression, Market Access, News, psychiatry, regulatory, Vraylar / February 23, 2022 February 23, 2022

AbbVie has filed with the FDA to extend the uses of its bipolar disorder therapy Vraylar to include adjunctive therapy for major depressive disorder, part of a plan to build sales of the drug to peak sales of $4 billion or more. The company is seeking approval from the US regulator for Vraylar (cariprazine) as …

AbbVie files Vraylar as add-on therapy for depression in US Read More »

Robert Califf confirmed as FDA commissioner in tight vote

FDA, Market Access, News, regulatory, Robert Califf, senior management / February 15, 2022 February 15, 2022

It’s official – President Joe Biden’s choice for FDA commissioner, Robert Califf, has been confirmed to the role after a narrow 50 to 46 vote in the Senate that relied on crossover Republican votes. That bipartisan support proved to be essential, with six Republican senators casting their lot for Califf to outweigh the five Democrats …

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Revenue cycle management checklist: improve experience & profits

Analytics, care management, claim denials, Claim Scrubber, claims management, ClaimSource, collections, Coverage Discovery, data, Featured, healthcare contract management solutions, Patient Access, Patient Collections, patient estimates, patient payment estimates, patient registration, Prior Authorization, prior-authorizations, regulatory, revenue cycle, Revenue Cycle Management, self-service solutions, state of patient access / February 9, 2022 February 9, 2022

Traditional revenue cycle management strategies are on shaky ground. Time-consuming manual processes are struggling under the burden of increasing patient volumes and patient financial responsibility. Clunky interfaces and disjointed systems fall short of evolving consumer expectations. Understanding and adapting to sweeping regulatory change is challenging for understaffed teams. On top of all of that, the …

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Drugmakers fined for colluding on NHS price of nausea drug

competition, News, NHS, pricing, regulatory, Sales and Marketing, UK / February 3, 2022 February 3, 2022

The UK Competition and Markets Authority has ruled that several drugmakers worked together to raise the NHS price of widely-used nausea drug prochlorperazine, fining them £35 million ($47.5 million). Alliance Pharmaceuticals, Lexon, Medreich, and former and current owners of Focus – Advanz and private equity group Cinven – were involved in an agreement to fix …

Drugmakers fined for colluding on NHS price of nausea drug Read More »

Reduce revenue loss with insurance Coverage Discovery

collections, Collections Optimization, Coverage Discovery, Featured, healthcare reimbursement, insurance coverage, No Surprises Act, patient experience, regulatory, regulatory updates, reimbursement, revenue cycle, Revenue Cycle Management / February 2, 2022 February 2, 2022

US hospitals have provided more than $702 billion in uncompensated care over the last two decades. To protect profits, healthcare organizations must be vigilant about finding any available insurance coverage for their patient’s care. But for many, recent regulatory changes and pandemic-related disruption have made navigating an already complex reimbursement landscape even more challenging. Checking …

Reduce revenue loss with insurance Coverage Discovery Read More »

MHRA seeks comments on post-Brexit clinical trial legislation

Brexit, Clinical Trials, MHRA, News, patients, regulatory / February 1, 2022 February 1, 2022

The MHRA has kicked off an eight-week consultation on proposals for changes to laws governing the conduct of clinical trials following the UK’s departure from the EU. The proposals stem from the passage of the Medicines and Medical Devices Act 2021 a year ago, shortly after the post-Brexit transition period ended, which has given the …

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EMA sets out proposals to reform EU’s clinical trials framework

Clinical Trials, EMA, European Commission, News, patients, regulatory / January 14, 2022 January 14, 2022

The European Commission, EMA and national regulators within the EU have launched an initiative to change the way clinical trials are designed and run in order to position the bloc as an international “focal point” for clinical research. The broad strokes of the proposals are laid out in a document entitled Accelerating Clinical Trials in …

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Applied Therapeutics slumps as FDA seeks more data on lead drug

Applied Therapeutics, galactosaemia, inherited disease, Market Access, News, rare disease, regulatory / January 3, 2022 January 3, 2022

Shares in Applied Therapeutics slumped today after it said it would have to delay a US filing for its lead drug programme AT-007, following an FDA request for more data. The New York-based biopharma company had been hoping to file AT007 in its lead indication galactosaemia in the third quarter of last year, but said …

Applied Therapeutics slumps as FDA seeks more data on lead drug Read More »

Biogen, Eisai Alzheimer’s drug lecanemab fast tracked by FDA

Aduhelm, alzheimer's disease, Biogen, Eisai, FDA, lecanemab, Market Access, neurology, News, regulatory / December 24, 2021 December 24, 2021

Biogen and Eisai head towards the end of the year with some much-needed good news in their Alzheimer’s programmes, as the FDA awards a fast-track designation to lecanemab, their follow-up to recently approved Aduhelm. The anti-amyloid drug, which also claimed a breakthrough tag from the FDA in July, is being developed for the treatment of …

Biogen, Eisai Alzheimer’s drug lecanemab fast tracked by FDA Read More »

No Surprises Act: Good Faith Estimate FAQ with an expert

Compliance, Featured, good faith estimate, good faith estimates, No Surprises Act, Price Transparency, regulatory / December 23, 2021 December 23, 2021

The No Surprises Act, effective Jan. 1, 2022, requires that healthcare providers include a “Good Faith Estimate” that covers all relevant codes and charges. This was established to increase price transparency for patients. For a summary of the No Surprises Act, read our previous blog. In our recent webinar, hosted on December 15, 2021, industry …

No Surprises Act: Good Faith Estimate FAQ with an expert Read More »

Japan looks set to follow EU in rejecting Alzheimer’s drug Aduhelm

Aduhelm, Biogen, Eisai, Japan, Market Access, News, regulatory / December 22, 2021 December 22, 2021

The Japanese regulator looks like it could follow the European Commission and reject approval of Biogen and Eisai’s Alzheimer’s disease therapy Aduhelm, leaving the FDA an outlier in its stance on the drug. An advisory committee to the Ministry of Health, Labour and Welfare (MHLW) has declined to approve Aduhelm (aducanumab), saying the data from …

Japan looks set to follow EU in rejecting Alzheimer’s drug Aduhelm Read More »

FDA sets April date for verdict on Incyte’s vitiligo drug

dermatology, FDA, Incyte, Market Access, News, Opzelura, regulatory, vitiligo / December 15, 2021 December 15, 2021

Incyte could be a few month away from a second FDA approval for its Opzelura cream that would make it the first medical treatment to re-pigment the skin of people with vitiligo. The FDA has kicked off a priority review of Opzelura (ruxolitinib), a JAK1/JAK2 inhibitor it approved to treat atopic dermatitis in September, and …

FDA sets April date for verdict on Incyte’s vitiligo drug Read More »

Califf breezes through Senate nomination hearing

FDA, Market Access, News, regulatory, Robert Califf, senior management / December 15, 2021 December 15, 2021

Robert Califf looks set to take a second term as FDA commissioner after navigating a relatively placid two-hour confirmation hearing in the US Senate, with just a few ripples of discontent over issues like opioid medicines and his ties to the pharma industry. President Joe Biden’s nominated candidate for the post told lawmakers that he …

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Intercept’s travails continue as it pulls NASH drug filing in EU

EU, hepatology, Intercept Pharmaceuticals, Market Access, NASH, News, regulatory / December 10, 2021 December 10, 2021

Already scrambling to refile its obeticholic acid (OCA) drug for non-alcoholic steatohepatitis (NASH) after the FDA rejected it last year, Intercept Pharma was hoping for more luck in Europe, but has now withdrawn its EU marketing application. Intercept had been hoping to extend the review period in the EU to allow time for results from …

Intercept’s travails continue as it pulls NASH drug filing in EU Read More »

FDA Update: BIMO – What Sponsors Need to Know

regulatory / December 6, 2021 December 6, 2021

The FDA’s Bioresearch Monitoring (BIMO) program is implemented to ensure the protection of the rights, safety, and welfare of human research subjects and to ensure the accuracy and reliability of clinical trial data. The FDA’s Office of Regulatory Affairs (ORA) Office of Bioresearch Monitoring conducts inspections related to FDA regulated research. The agency recently updated …

FDA Update: BIMO – What Sponsors Need to Know Read More »

BeyondSpring faces a tough winter as FDA rejects its lead drug

BeyondSpring, chemotherapy, FDA, News, Oncology, Plinabulin, regulatory / December 1, 2021 December 1, 2021

BeyondSpring’s hopes of an early Christmas gift from the FDA were dashed today after the regulator said it was unable to approve the company’s lead drug plinabulin as a treatment for low white blood cell counts caused by cancer chemotherapy. In a complete response letter (CRL), the FDA said that it would need a second …

BeyondSpring faces a tough winter as FDA rejects its lead drug Read More »

Jazz’ Sunosi (solriamfetol) Receives NICE Recommendation for the Treatment of Excessive Daytime Sleepiness Caused by Narcolepsy

Excessive Daytime Sleepiness, Jazz, narcolepsy, NICE, Recommendation, regulatory, solriamfetol, Sunosi / November 19, 2021 November 19, 2021

Shots: NICE has issued a positive Final Appraisal Document which recommends Sunosi (75/150mg, qd) as an option treatment for adults with EDS caused by narcolepsy with/out cataplexy. The therapy will be available in England and Wales The recommendation is based on the P-III TONES studies to evaluate Sunosi vs PBO in adults patients with EDS. …

Jazz’ Sunosi (solriamfetol) Receives NICE Recommendation for the Treatment of Excessive Daytime Sleepiness Caused by Narcolepsy Read More »

Antengene’s Selinexor (ATG-010) Receives NMPA’s IND Approval for P-I/II SWATCH Study to Treat Non-Hodgkin Lymphoma

(Selinexor), Antengene, approval, ATG-010, IND, NMPA, non-Hodgkin lymphoma, P-I/II, regulatory, SWATCH Study / November 19, 2021 November 19, 2021

Shots: The NMPA has approved a P-I/II SWATCH dose-escalation & dose-expansion study that evaluates the safety, tolerability, and preliminary efficacy of selinexor in combination with the R2 regimen of lenalidomide + rituximab for rrDLBCL and rriNHL who are not eligible for HDC or ASCT. The study was conducted at 10 centers across China The 1EPs …

Antengene’s Selinexor (ATG-010) Receives NMPA’s IND Approval for P-I/II SWATCH Study to Treat Non-Hodgkin Lymphoma Read More »

Merck’s Keytruda (pembrolizumab) Receives the US FDA’s Approval as a Adjuvant Therapy for Renal Cell Carcinoma

approval, FDA, KEYTRUDA, Merck, pembrolizumab, regulatory, Renal Cell Carcinoma, US / November 19, 2021 November 19, 2021

Shots: The approval is based on the P-III KEYNOTE-564 trial evaluating Keytruda (200mg, IV, q3w for 1yrs.) vs PBO in a ratio (1:1) in 994 patients with intermediate-high or high risk of recurrence of RCC or M1 NED, following nephrectomy and resection of metastatic lesions The results showed an improvement in DFS, 32% reduction in …

Merck’s Keytruda (pembrolizumab) Receives the US FDA’s Approval as a Adjuvant Therapy for Renal Cell Carcinoma Read More »

GSK’s Nucala (mepolizumab) Receives the EC’s Approval for the Treatment of Eosinophil-Driven Diseases

approval, EC, Eosinophil-Driven Diseases, GSK, mepolizumab, Nucala, regulatory / November 18, 2021 November 18, 2021

Shots: The approvals are based on the clinical trials to evaluate mepolizumab vs PBO when added to SoC in patients with HES, EGPA & CRSwNP In HES, patients experienced a HES flare (28% vs 56%) @ 32wks. The therapy increased both accrued time in remission & the proportion of patients who achieved remission in patients …

GSK’s Nucala (mepolizumab) Receives the EC’s Approval for the Treatment of Eosinophil-Driven Diseases Read More »

AstraZeneca’s Lokelma Receives Fast Track Designation for the Treatment of Chronic Haemodialysis with Hyperkalaemia

AstraZeneca, Chronic Haemodialysis, Fast Track Designation, hyperkalaemia, Lokelma, regulatory / November 18, 2021 November 18, 2021

Shots: The designation is based on the ongoing P-III DIALIZE-Outcomes trial to evaluate the effect of Lokelma on arrhythmia-related CV outcomes in patients with chronic hemodialysis with recurrent HK at 300 sites across the US, Canada, Russia, EU, Asia & South America The therapy is designed to reduce serious adverse CV outcomes. The results are …

AstraZeneca’s Lokelma Receives Fast Track Designation for the Treatment of Chronic Haemodialysis with Hyperkalaemia Read More »

AbbVie’s Skyrizi (risankizumab) Receives EC’s Approval for the Treatment of Active Psoriatic Arthritis

regulatory / November 17, 2021 November 17, 2021

Shots: The approval is based on KEEPsAKE-1 & 2 studies to evaluate Skyrizi (150mg) vs PBO in patients with active PsA who had an inadequate response or were intolerant to biologic therapy & & DMARDs The trials meet its 1EPs i.e., patients achieved ACR20 response @24wks. (57.3% & 51.3%) vs (33.5% & 26.5%). The 2EPs …

AbbVie’s Skyrizi (risankizumab) Receives EC’s Approval for the Treatment of Active Psoriatic Arthritis Read More »

Biogen’s Vumerity (diroximel fumarate) Receives the EC’s Approval for the Treatment of Relapsing-Remitting Multiple Sclerosis

approval, Biogen, Diroximel Fumarate, EC, regulatory, Vumerity / November 17, 2021 November 17, 2021

Shots: The approval is based on the data from PK bridging studies and P-III EVOLVE-MS-2 study to evaluate the GI tolerability of Vumerity vs Tecfidera in patients with relapsing-remitting MS In the EVOLVE-MS-2 study, the therapy demonstrated a low rate of overall treatment discontinuation due to GI tolerability (1.6% vs 6%). Additionally, flushing was reported …

Biogen’s Vumerity (diroximel fumarate) Receives the EC’s Approval for the Treatment of Relapsing-Remitting Multiple Sclerosis Read More »

Celltrion’s Regdanvimab Receives the EC’s Approval for the Treatment of COVID-19

approval, Celltrion, COVID-19, EC, Regdanvimab, regulatory / November 16, 2021 November 16, 2021

Shots: The EC has granted marketing authorisation for Celltrion’s regdanvimab in adults with COVID-19 who do not require supplemental oxygen & are at high risk of progressing to severe COVID-19 The approval is based on the P-III trial to evaluate the efficacy & safety of regdanvimab in 1315+ patients with COVID-19 across 13 countries including …

Celltrion’s Regdanvimab Receives the EC’s Approval for the Treatment of COVID-19 Read More »

Coherus and Junshi’s Toripalimab Receive the US FDA’s Orphan Drug Designation for the Treatment of Esophageal Cancer

Coherus, Esophageal Cancer, FDA, Junshi, Orphan Drug Designation, regulatory, Toripalimab, US / November 16, 2021 November 16, 2021

Shots: The designation is based on the results of the P-III JUPITER-06 clinical trial that evaluates toripalimab + CT vs CT alone in patients with advanced or metastatic ESCC The study met the co-primary EPs i.e., improvements in PFS & OS for patients treated with the toripalimab + CT compared to CT alone The company …

Coherus and Junshi’s Toripalimab Receive the US FDA’s Orphan Drug Designation for the Treatment of Esophageal Cancer Read More »

Vertex’s Kaftrio (ivacaftor/tezacaftor/elexacaftor) + Ivacaftor Receives CHMP’s Positive Opinion for the Treatment of Cystic Fibrosis in Children Aged 6 to 11

CHMP, Cystic fibrosis, elexacaftor, Ivacaftor, Kaftrio, regulatory, tezacaftor, Vertex / November 15, 2021 November 15, 2021

Shots: The EMA’s CHMP has adopted a positive opinion for the label extension of Kaftrio in combination with ivacaftor to treat CF in patients aged 6 to11 yrs. who have at least one F508del mutation in a CFTR gene The combination therapy showed improvements in lung function, sweat chloride & respiratory symptoms, safety & tolerability …

Vertex’s Kaftrio (ivacaftor/tezacaftor/elexacaftor) + Ivacaftor Receives CHMP’s Positive Opinion for the Treatment of Cystic Fibrosis in Children Aged 6 to 11 Read More »

Amgen’s Lumykras (sotorasib) Receives CHMP’s Positive Opinion for the Treatment of KRAS G12C-Mutated Advanced Non-Small Cell Lung Cancer

Amgen, CHMP, KRAS G12C-Mutated, Lumykras, Non-Small Cell Lung Cancer, regulatory, Sotorasib / November 15, 2021 November 15, 2021

Shots: The EMA’s CHMP has adopted a positive opinion recommending the conditional marketing authorization for Lumykras to treat advanced NSCLC with KRAS G12C mutation & who have progressed after 1 prior line of systemic therapy The CHMP opinion is based on the P-II CodeBreaK 100 trial to evaluate sotorasib (960mg, qd, PO) in 126 patients …

Amgen’s Lumykras (sotorasib) Receives CHMP’s Positive Opinion for the Treatment of KRAS G12C-Mutated Advanced Non-Small Cell Lung Cancer Read More »

Novo Nordisk’s Wegovy (semaglutide) Receives CHMP’s Recommendation for Approval to Treat Obesity

approval, CHMP, Novo Nordisk, obesity, regulatory, semaglutide, Wegovy / November 12, 2021 November 12, 2021

Shots: The EMA’s CHMP has adopted a positive opinion recommending the marketing authorization for Wegovy in adults with obesity in chronic weight management in adults with obesity The approval is based on P-IIIa (STEP) study evaluating Wegovy (2.4mg injection, qw) in ~4,500 adults with obesity or overweight with one weight-related comorbidity. The results showed 17-18% …

Novo Nordisk’s Wegovy (semaglutide) Receives CHMP’s Recommendation for Approval to Treat Obesity Read More »

Celltrion’s CT-P59 (regdanvimab) Receives CHMP’s Positive Opinion for the Treatment of COVID-19

Celltrion, CHMP, COVID-19, CT-P59, Regdanvimab, regulatory / November 12, 2021 November 12, 2021

Shots: The EMA’s CHMP has issued a positive opinion recommending the marketing authorization for regdanvimab (CT-P59) in adults with COVID-19 who do not require supplemental oxygen & are at increased risk of progressing to severe COVID-19 The CHMP opinion was based on the P-III clinical trial to evaluate the efficacy and safety of regdanvimab in …

Celltrion’s CT-P59 (regdanvimab) Receives CHMP’s Positive Opinion for the Treatment of COVID-19 Read More »

(Webinar) FDA Update: BIMO – What Sponsors Need to Know

regulatory / November 8, 2021 November 8, 2021

(Webinar) FDA Update: BIMO – What Sponsors Need to Know Read More »

Amylyx Submits NDA to the US FDA for AMX0035 to Treat Amyotrophic Lateral Sclerosis

ALS, AMX0035, Amylyx, FDA, NDA, regulatory, US / November 3, 2021 November 3, 2021

Shots: The NDA is based on CENTAUR trial evaluates AMX0035 vs PBO in 137 patients with ALS The results showed that patients receiving AMX0035 had a significant functional decline at the end of the 6mos. as measured by the ALSFRS-R. In an interim survival analysis @3yrs., 44% lower risk of death, median survival duration (25.0mos. …

Amylyx Submits NDA to the US FDA for AMX0035 to Treat Amyotrophic Lateral Sclerosis Read More »

Antengene’s Receives NMPA’s IND Approval for P-Ib MATCH Study of ATG-008 (onatasertib) ATG-010 (selinexor) to Treat Diffuse Large B-Cell Lymphoma

(Selinexor), Antengene, approval, ATG-008, ATG-010, diffuse large B-cell lymphoma, IND, MATCH Study, NMPA, onatasertib, P-Ib, regulatory / November 2, 2021 November 2, 2021

Shots: The NMPA has approved an IND application for P-Ib MATCH, dose-escalation study to evaluate the safety of ATG-008 + ATG-010 in patients with r/r DLBCL. The study will be conducted at ten centers across China, including its primary trial center at Sun Yat-Sen University Cancer Center The 1EPs of the study include MTD and …

Antengene’s Receives NMPA’s IND Approval for P-Ib MATCH Study of ATG-008 (onatasertib) ATG-010 (selinexor) to Treat Diffuse Large B-Cell Lymphoma Read More »

Junshi and Coherus Report the US FDA Acceptance of BLA for Toripalimab to Treat Nasopharyngeal Carcinoma

BLA, Coherus, FDA, Junshi, Nasopharyngeal Carcinoma, regulatory, Toripalimab, US / November 2, 2021 November 2, 2021

Shots: The US FDA has accepted the BLA for toripalimab + CT (gemcitabine & cisplatin) & toripalimab as monothx. to treat advanced recurrent or metastatic NPC after platinum-containing CT. The US FDA has granted PR designation of toripalimab’ BLA with an anticipated PDUFA date on Apr’22 The BLA is based on the P-II POLARIS-02 & …

Junshi and Coherus Report the US FDA Acceptance of BLA for Toripalimab to Treat Nasopharyngeal Carcinoma Read More »

Incyte Reports the US FDA’s Acceptance of NDA for Parsaclisib to Treat R/R Non-Hodgkin Lymphomas

FDA, Incyte, NDA, Parsaclisib, R/R Non-Hodgkin Lymphomas, regulatory, US / November 1, 2021 November 1, 2021

Shots: The submission is based on P-II studies i.e., CITADEL-203/204/205 that evaluate parsaclisib in patients with r/r FL, MZL & MCL. The anticipated PDUFA date for parsaclisib in adult patients with r/r FL is Aug 30, 2022. The results from the studies showed that Parsaclisib was generally well-tolerated with a manageable safety profile The FDA …

Incyte Reports the US FDA’s Acceptance of NDA for Parsaclisib to Treat R/R Non-Hodgkin Lymphomas Read More »

Novartis’ Scemblix (asciminib) Receives the US FDA’s Approval for the Treatment of Chronic Myeloid Leukemia

regulatory / November 1, 2021 November 1, 2021

Shots: The approval is based on P-III ASCEMBL & P-I study that evaluates Scemblix in patients with Ph+ CML-CP with T315I mutation who had experienced resistance or intolerance to two TKIs The P-III ASCEMBL trial showed an MMR rate (25% vs 13%) @24wks. & patients who discontinued treatment due to AEs (7% vs 25%). An …

Novartis’ Scemblix (asciminib) Receives the US FDA’s Approval for the Treatment of Chronic Myeloid Leukemia Read More »

ADC Reports EMA’s Validation of MAA for Zynlonta to Treat R/R Diffuse Large B-Cell Lymphoma

(R/R) Diffuse Large B-Cell Lymphoma, ADC, EMA, MAA, Pharma, regulatory, Zynlonta / October 29, 2021 October 29, 2021

Shots: The MAA is based on the P-II LOTIS-2 study evaluating Zynlonta (CD19-directed ADC) in 145 patients with r/r DLBCL following two or more prior lines of systemic therapy The results demonstrated 48.3% ORR; 24.1% CR rate; 24.1% PR rate; patients had a median time to response of 1.3mos. & m-DoR was 13.4mos. With the …

ADC Reports EMA’s Validation of MAA for Zynlonta to Treat R/R Diffuse Large B-Cell Lymphoma Read More »

Impact Receives US FDA’s IND Clearance for IMP9064 to Treat Solid Tumor

FDA, IMP9064, Impact, IND, regulatory, solid tumor, US / October 29, 2021 October 29, 2021

Shots: The company will initiate the P-I/II dose-escalation & expansion study to evaluate the safety, PK & anti-tumor activities of IMP9064 as monothx. & in combination with Senaparib in patients with advanced solid tumors In preclinical studies, IMP9064 was a highly potent ATR inhibitor & selective against other kinases & showed high activities in several …

Impact Receives US FDA’s IND Clearance for IMP9064 to Treat Solid Tumor Read More »

Novartis Reports the US FDA and EMA’s Acceptance of sBLA for Kymriah (tisagenlecleucel) and Granted Priority Review to Treat R/R Follicular Lymphoma

EMA, FDA, Kymriah, Novartis, Priority Review, R/R Follicular Lymphoma, regulatory, sBLA, tisagenlecleucel, US / October 29, 2021 October 29, 2021

Shots: The submissions are based on the P-II ELARA trial evaluates the efficacy and safety of Kymriah in adult patients with r/r FL after at least two prior therapies over 30 sites in 12 countries globally The trial met its 1EPs i.e., the therapy showed a robust response in heavily pretreated patients & the safety …

Novartis Reports the US FDA and EMA’s Acceptance of sBLA for Kymriah (tisagenlecleucel) and Granted Priority Review to Treat R/R Follicular Lymphoma Read More »

Kite’s Tecartus (brexucabtagene autoleucel) Receives Health Canada’s Approval for the Treatment of Relapsed or Refractory Mantle Cell Lymphoma

approval, brexucabtagene autoleucel, Health Canada, Kite, mantle cell lymphoma, regulatory, Tecartus / October 29, 2021 October 29, 2021

Shots: Health Canada has granted a NOC based on the P-II ZUMA-2 trial that evaluates Tecartus in 74 adult patients with r/r MCL who had previously received anthracycline or bendamustine containing CT, an anti-CD20 Ab therapy & BTK inhibitor The results demonstrated an ORR of 91%; patients achieving a CR (65%) as assessed by an …

Kite’s Tecartus (brexucabtagene autoleucel) Receives Health Canada’s Approval for the Treatment of Relapsed or Refractory Mantle Cell Lymphoma Read More »

Incyte Reports EMA’s Validation of MAA for Ruxolitinib Cream to Treat Vitiligo

EMA, Incyte, MAA, regulatory, Ruxolitinib Cream, vitiligo / October 29, 2021 October 29, 2021

Shots: The MAA is based on the P-III TRuE-V clinical program that consists of 2 P-III studies ie., TRuE-V1 & V2 evaluating ruxolitinib cream in 600+ patients aged ≥12yrs. with vitiligo The therapy demonstrated improvements in facial & total body repigmentation @ 24wks. of treatment, patients treated with ruxolitinib cream did not report site reactions …

Incyte Reports EMA’s Validation of MAA for Ruxolitinib Cream to Treat Vitiligo Read More »

Pfizer and BioNTech’s COVID-19 Vaccine Receive the US FDA’s Advisory Committee Vote Supporting EUA Against COVID-19

Advisory Committee, BioNTech, COVID-19, Covid-19 Vaccine, EUA, FDA, Pfizer, regulatory, US / October 27, 2021 October 27, 2021

Shots: The US FDA’s VRBPAC voted 17 to 0 with 1 abstention in favor of recommending the EUA for COVID-19 vaccine in children aged 5 to <12yrs. with COVID-19 The committee reviewed the P-II/III trial for the COVID-19 vaccine which showed a 90.7% vaccine efficacy in patients without prior SARS-CoV-2 infection @7 Days after 2nd …

Pfizer and BioNTech’s COVID-19 Vaccine Receive the US FDA’s Advisory Committee Vote Supporting EUA Against COVID-19 Read More »

Merck and Ridgeback Report the Initiation of EMA Rolling Review of Molnupiravir for the Treatment of COVID-19

COVID-19, EMA, Merck, molnupiravir, regulatory, Ridgeback / October 25, 2021 October 25, 2021

Shots: The submission is based on the P-III MOVe-OUT trial that evaluates molnupiravir (800mg, bid) in non-hospitalized adult patients with COVID-19 At the interim analysis, the therapy showed a ~50% reduction in risk of hospitalization or death; patients were hospitalized (7.3% vs 14.1%) through 29Day following randomization, no deaths were reported in patients who received …

Merck and Ridgeback Report the Initiation of EMA Rolling Review of Molnupiravir for the Treatment of COVID-19 Read More »

Genentech’s Susvimo Receives the US FDA’s Approval for the Treatment of Wet Age-Related Macular Degeneration

approval, FDA, Genentech, regulatory, Susvimo, US, Wet Age-Related Macular Degeneration / October 25, 2021 October 25, 2021

Shots: The approval is based on P-III Archway study evaluates Susvimo (100 mg/mL, IV, q3mos.) via ocular implant vs ranibizumab (0.5mg, IV) in 415 patients with wet AMD prior responded to 2 anti- VEGF The results showed that the therapy achieved & maintained vision gains equivalent to ranibizumab @ 36 & 40wks.; 1.6% of patients …

Genentech’s Susvimo Receives the US FDA’s Approval for the Treatment of Wet Age-Related Macular Degeneration Read More »

Pfizer’s Prevnar 20 (Pneumococcal 20-Valent Conjugate Vaccine) Receives the CDC’s Advisory Committee Recommendation for Invasive Disease and Pneumonia

Advisory Committee, CDC, Invasive Disease, Pfizer, Pneumococcal 20-Valent Conjugate Vaccine, pneumonia, PREVNAR 20, regulatory / October 21, 2021 October 21, 2021

Shots: The CDC’s ACIP has voted to recommend Prevnar 20 (Pneumococcal 20-valent Conjugate Vaccine) to protect adults against invasive disease and pneumonia caused by the 20 S. pneumoniae serotypes The ACIP has recommended one dose of Prevnar 20 for adults aged ≥65 & for ≥19yrs. with risk conditions who have not previously received a pneumococcal …

Pfizer’s Prevnar 20 (Pneumococcal 20-Valent Conjugate Vaccine) Receives the CDC’s Advisory Committee Recommendation for Invasive Disease and Pneumonia Read More »

Regeneron and Sanofi’s Dupixent (dupilumab) Receive the US FDA’s Approval as an Add-On Maintenance Treatment to Treat Moderate-To-Severe Asthma

approval, Asthma, dupilumab, Dupixent, FDA, Regeneron, regulatory, Sanofi, US / October 21, 2021 October 21, 2021

Shots: The P-III LIBERTY ASTHMA VOYAGE Trial evaluates Dupixent (100/200mg, q2w) + SoC in 408 children aged 6-11yrs. with uncontrolled mod. to sev. asthma The results showed a 65% avg. reduction in the rate of severe asthma attacks @1yrs.; improvement in lung function by 5.32% points @ 12wks. & as early as 2wks. & sustained …

Regeneron and Sanofi’s Dupixent (dupilumab) Receive the US FDA’s Approval as an Add-On Maintenance Treatment to Treat Moderate-To-Severe Asthma Read More »

Sanofi’s Dupixent (dupilumab) Receives NICE Recommendation for the Treatment of Severe Asthma

dupilumab, Dupixent, NICE, regulatory, Sanofi, severe asthma / October 19, 2021 October 19, 2021

Shots: The NICE has issued a final appraisal determination (FAD) which recommends Dupixent (dupilumab) as an add-on maintenance treatment for severe asthma The NICE recommendation is based on a LIBERTY ASTHMA QUEST trial evaluating Dupixent vs PBO in patients with severe asthma with high exacerbation rates and high biomarkers indicating type 2 inflammation The results …

Sanofi’s Dupixent (dupilumab) Receives NICE Recommendation for the Treatment of Severe Asthma Read More »

Gilead’s Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide) Low-Dose Tablet Receives the US FDA’s Approval for Expanded Indication to Treat HIV-1

approval, Bictegravir, Biktarvy, Emtricitabine, FDA, Gilead, HIV-1, regulatory, Tenofovir Alafenamide, US / October 19, 2021 October 19, 2021

Shots: The approval is based on cohort 3 of a P-II/III study that evaluates Biktarvy (bictegravir 30mg/emtricitabine 120mg/tenofovir alafenamide 15mg) in 22 Pediatric patients with HIV-1 who are virologically suppressed or new to antiretroviral therapy The results showed that Biktarvy low-dose tablets were found to be effective & well-tolerated @24wks. After switching to Biktarvy, 91% …

Gilead’s Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide) Low-Dose Tablet Receives the US FDA’s Approval for Expanded Indication to Treat HIV-1 Read More »

Boehringer Ingelheim’s Cyltezo (biosimilar, adalimumab) Receives the US FDA’s sBLA Approval as the 1st Interchangeable Biosimilar for Multiple Chronic Inflammatory Diseases

(adalimumab), approval, Biosimilar, Biosimilars, Boehringer Ingelheim, Cyltezo, FDA, inflammatory diseases, regulatory, sBLA, US / October 18, 2021 October 18, 2021

Shots: The approval is based on the P-III VOLTAIRE-X trial evaluates the effects of multiple switches b/w Humira and Cyltezo in patients with multiple chronic inflammatory diseases The study showed that Cyltezo was equivalent to Humira & demonstrated that switching several times b/w Cyltezo and Humira resulted in no clinical differences in PK, efficacy, immunogenicity, …

Boehringer Ingelheim’s Cyltezo (biosimilar, adalimumab) Receives the US FDA’s sBLA Approval as the 1st Interchangeable Biosimilar for Multiple Chronic Inflammatory Diseases Read More »

AbbVie’ Skyrizi (risankizumab) Receives CHMP’s Positive Opinion for the Treatment of Adults with Active Psoriatic Arthritis in the EU

AbbVie, Active Psoriatic Arthritis, CHMP, EU, regulatory, risankizumab, Skyrizi / October 18, 2021 October 18, 2021

Shots: The opinion was based on the P-III KEEPsAKE-1 & 2 studies evaluate Skyrizi (150mg, SC) vs PBO in adults with active PsA who had an inadequate response or were intolerant to biologic therapy & non-biologic DMARDs The trial met its 1EPs of ACR20 response @24wks. The 2EPs also met that include improvements in clinical …

AbbVie’ Skyrizi (risankizumab) Receives CHMP’s Positive Opinion for the Treatment of Adults with Active Psoriatic Arthritis in the EU Read More »

Genentech’s Tecentriq Receives the US FDA’s Approval as Adjuvant Treatment for Early Non-Small Cell Lung Cancer

approval, FDA, Genentech, Non-Small Cell Lung Cancer, regulatory, Tecentriq, US / October 18, 2021 October 18, 2021

Shots: The approval is based on P-III IMpower010 study Tecentriq vs BSC in 1005 patients in a ratio (1:1) with Stage IB-IIIA NSCLC, following surgical resection and up to 4 cycles of adjuvant cisplatin-based CT The results showed a 34% reduction in risk of disease recurrence or death in people with Stage II-IIIA NSCLC whose …

Genentech’s Tecentriq Receives the US FDA’s Approval as Adjuvant Treatment for Early Non-Small Cell Lung Cancer Read More »

Califf re-joining FDA would be positive for industry; analyst

FDA, Market Access, News, regulatory, Robert Califf, senior management / October 17, 2021 October 17, 2021

With rumours growing that that ex-FDA head Robert Califf looks set to receive President Joe Biden’s nomination to be the next FDA commissioner, at least one analyst thinks the appointment would be a positive for the biopharma industry. Califf was appointed to the helm of the FDA under the Obama administration in 2016, and picked …

Califf re-joining FDA would be positive for industry; analyst Read More »

Bristol Myers Squibb’s Zeposia (ozanimod) Receives CHMP’s Positive Opinion for Adult Patients with Moderately to Severely Active Ulcerative Colitis

(ozanimod), Adult Patients with Moderately to Severely Active Ulcerative Colitis, Bristol-Myers Squibb, CHMP, Positive Opinion, regulatory, Zeposia / October 15, 2021 October 15, 2021

Shots: The CHMP’s recommendation is based on P-III True North study evaluating Zeposia as an induction and maintenance therapy vs PBO in adult patients with moderately to severely active UC The study demonstrated improvements across all 1EPs and 2EPs including clinical remission, clinical response, endoscopic improvement and endoscopic histologic mucosal improvement @10 & 52wks. If …

Bristol Myers Squibb’s Zeposia (ozanimod) Receives CHMP’s Positive Opinion for Adult Patients with Moderately to Severely Active Ulcerative Colitis Read More »

Regeneron Reports the US FDA’s Acceptance of Priority Review for REGEN-COV for the Treatment and Prophylaxis of COVID-19

Acceptance, COVID-19, FDA, Priority Review, REGEN-COV, Regeneron, regulatory, US / October 14, 2021 October 14, 2021

Shots: The US FDA has accepted for priority review a BLA for REGEN-COV (casirivimab and imdevimab) to treat COVID-19 in non-hospitalized patients and as prophylaxis in certain individuals The BLA is based on P-III study assessing efficacy & safety of REGEN-COV to treat non-hospitalized patients already infected with SARS-CoV-2, and to prevent symptomatic infection in …

Regeneron Reports the US FDA’s Acceptance of Priority Review for REGEN-COV for the Treatment and Prophylaxis of COVID-19 Read More »

Asieris Receives NMPA’s IND Approval to Evaluate APL-1202 + Tislelizumab as Neoadjuvant Therapy for MIBC Patients

APL-1202, Asieris, IND Approval, MIBC, Neoadjuvant Therapy, NMPA, regulatory, Tislelizumab / October 14, 2021 October 14, 2021

Shots: NMPA’s CDE has approved the IND application of oral APL-1202 in combination with BeiGene’s tislelizumab as neoadjuvant therapy in patients with muscle invasive bladder cancer. The US FDA has cleared the IND of the therapy in Jun’2021 The P-I/II study will evaluate the safety in MIBC patients, to determine RP2D and to assess efficacy …

Asieris Receives NMPA’s IND Approval to Evaluate APL-1202 + Tislelizumab as Neoadjuvant Therapy for MIBC Patients Read More »

Novartis Reports the US FDA and EMA Filing Acceptance of Beovu for Patients with Diabetic Macular Edema

Beovu, Diabetic Macular Edema, EMA, FDA, Novartis, regulatory, US / October 14, 2021 October 14, 2021

Shots: The US FDA has accepted the sBLA and EMA has validated the type-II variation application for Beovu (brolucizumab, 6mg) for the treatment of DME. Additionally, the PMDA has accepted an application for Beovu for the same indication The application is based on P-III KESTREL and KITE studies, which met their 1EPs of non-inferiority in …

Novartis Reports the US FDA and EMA Filing Acceptance of Beovu for Patients with Diabetic Macular Edema Read More »

Merck’s Keytruda (pembrolizumab) Receives the US FDA’s Approval for Second Indication in Cervical Cancer

approval, cervical cancer, FDA, KEYTRUDA, Merck, pembrolizumab, regulatory, Second Indication, US / October 14, 2021 October 14, 2021

Shots: The US FDA has approved Keytryda + CT, with/out bevacizumab, for the treatment of patients with persistent, recurrent or metastatic cervical cancer whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test The approval is based on the P-III KEYNOTE-826 trial that demonstrated superior OS and PFS, ORR (68% vs 50%), mDOR …

Merck’s Keytruda (pembrolizumab) Receives the US FDA’s Approval for Second Indication in Cervical Cancer Read More »

PharmaTher’s Ketamine Receives the US FDA’s Orphan Drug Designation for Complex Regional Pain Syndrome

Complex Regional Pain Syndrome, FDA, ketamine, Orphan Drug Designation, PharmaTher, regulatory, US / October 14, 2021 October 14, 2021

Shots: The US FDA has granted ODD to the Ketamine for the treatment of complex regional pain syndrome (CRPS) which is a rare chronic pain and inflammatory condition following an injury to a limb The ODD follows the US FDA’s ODD of ketamine for the treatment of ALS, granted on Aug 04, 2021 Ketamine acts …

PharmaTher’s Ketamine Receives the US FDA’s Orphan Drug Designation for Complex Regional Pain Syndrome Read More »

Eli Lilly’s Verzenio (abemaciclib) Receives the US FDA’s Approval for People with HR+ HER2- High Risk Early Breast Cancer

regulatory / October 13, 2021 October 13, 2021

Shots: The US FDA has approved Verzenio + ET for the adjuvant treatment of adult patients with HR+, HER2-, node-positive, EBC at high risk of recurrence and a Ki-67 score of ≥20% as determined by an FDA-approved test The approval is based on subgroup analyses of P-III monarchE trial with additional follow-up, conducted post-hoc. In …

Eli Lilly’s Verzenio (abemaciclib) Receives the US FDA’s Approval for People with HR+ HER2- High Risk Early Breast Cancer Read More »

Sanofi’s Sarclisa Combination Regimen Receives Health Canada’s Approval for Multiple Myeloma

approval, Combination Regimen, Health Canada, multiple myeloma, regulatory, Sanofi, Sarclisa / October 13, 2021 October 13, 2021

Shots: The approval is based on P-III IKEMA study assessing Sarclisa in combination with carfilzomib and dexamethasone (Kd) in 302 patients with relapsed MM who have received 1 to 3 prior lines of therapy across 69 centers spanning 16 countries The addition of Kd in Sarclisa leads to a 47% reduction in the risk of …

Sanofi’s Sarclisa Combination Regimen Receives Health Canada’s Approval for Multiple Myeloma Read More »

Merck and Ridgeback Report EUA Submission to the US FDA for Molnupiravir to Treat COVID-19

COVID-19, EUA, FDA, Merck, molnupiravir, regulatory, Ridgeback, US / October 11, 2021 October 11, 2021

Shots: The P-III MOVe-OUT trial evaluates molnupiravir vs PBO in non-hospitalized adult patients with COVID-19 with 1 risk factor associated with poor disease outcomes & symptom onset within 5 days before randomization The interim analysis showed ~50% reduction in risk of hospitalization or death; patients who received therapy were either hospitalized or died @29 Days …

Merck and Ridgeback Report EUA Submission to the US FDA for Molnupiravir to Treat COVID-19 Read More »

Genentech’s Gantenerumab Receives the US FDA’s Breakthrough Therapy Designation for the Treatment of Alzheimer’s Disease

alzheimer's disease, Breakthrough Therapy Designation, FDA, gantenerumab, Genentech, regulatory, US / October 11, 2021 October 11, 2021

Shots: The designation is based on the data from an ongoing SCarlet RoAD, Marguerite RoAD OLE trials along with other studies evaluating gantenerumab (anti-amyloid beta Ab) in patients with AD, demonstrated a reduction in brain amyloid plaque The studies have been included in the optimized design of two ongoing P-III trials, GRADUATE 1 & 2 …

Genentech’s Gantenerumab Receives the US FDA’s Breakthrough Therapy Designation for the Treatment of Alzheimer’s Disease Read More »

Janssen Report Submission of sBLA to the US FDA for the Approval of Stelara (ustekinumab) to Treat Juvenile Psoriatic Arthritis

FDA, Janssen, Juvenile Psoriatic Arthritis, regulatory, sBLA, Stelara, US, ustekinumab / October 11, 2021 October 11, 2021

Shots: The company has submitted an sBLA to the US FDA for an expanded approval of Stelara (ustekinumab) to treat pediatric patients ≥aged 5yrs. with jPsA The submission is based on the extrapolation of data from the nine studies evaluating the efficacy and tolerability of Stelara in 3,997 patients across both adults with active PsA …

Janssen Report Submission of sBLA to the US FDA for the Approval of Stelara (ustekinumab) to Treat Juvenile Psoriatic Arthritis Read More »

AbbVie’s Rinvoq (upadacitinib) Receives the Health Canada Approval for the Treatment of Moderate to Severe Atopic Dermatitis

AbbVie, approval, Atopic Dermatitis, Health Canada, regulatory, Rinvoq, Upadacitinib / October 8, 2021 October 8, 2021

Shots: The approval is based on the P-III clinical trial program including Measure Up 1/2/AD Up that evaluates the efficacy & safety of Rinvoq (15/30 mg, qd) as monothx. with TCS vs PBO in 2500+ adults & adolescents with AD In all three studies, the co-primary EPs showed 75% improvement in EASI 75 and vIGA-AD …

AbbVie’s Rinvoq (upadacitinib) Receives the Health Canada Approval for the Treatment of Moderate to Severe Atopic Dermatitis Read More »

Takeda Receives the US FDA Advisory Committee’s Recommendation for TAK-620 (maribavir) to Treat CMV Infection in Post-Transplant Recipients

Advisory Committee, CMV Infection, FDA, maribavir, regulatory, TAK-620, Takeda, US / October 8, 2021 October 8, 2021

Shots: The US FDA’s AMDAC voted to recommend the use of maribavir (TAK-620) to treat refractory CMV infection and disease with/out genotypic resistance to ganciclovir, valganciclovir, foscarnet, or cidofovir in transplant recipients The recommendations were based on the results from the P-II & III TAK-620-303 (SOLSTICE) trials of maribavir while the NDA submission of maribavir …

Takeda Receives the US FDA Advisory Committee’s Recommendation for TAK-620 (maribavir) to Treat CMV Infection in Post-Transplant Recipients Read More »

Novartis’s Adakveo (crizanlizumab) Receives NICE Recommendation as a New Therapy for Sickle Cell Disease

Adakveo, crizanlizumab, NICE, Novartis, Recommendation, regulatory / October 6, 2021 October 6, 2021

Shots: The NICE has recommended Novartis’ Adakveo (crizanlizumab) as a new treatment option to prevent recurrent sickle cell crises in patients aged ≥16 Additionally, patients will now have access to this treatment option on the NHS. The clinical evidence demonstrated that the people treated with crizanlizumab have significantly fewer sickle cell crises in a year …

Novartis’s Adakveo (crizanlizumab) Receives NICE Recommendation as a New Therapy for Sickle Cell Disease Read More »

Johnson & Johnson Reports EUA Submission to the US FDA for Supporting the Booster of its Single Shot COVID-19 Vaccine Candidate

Covid-19 Vaccine Candidate, EUA, FDA, Johnson & Johnson, regulatory, Single Shot, US / October 6, 2021 October 6, 2021

Shots: The submission is based on the P-III ENSEMBLE 2 study that evaluates the booster dose COVID-19 vaccine in adults aged ≥18yrs with COVID-19 The results demonstrated a 94% protection against symptomatic COVID-19 in the US for booster dose @56 days after primary dose & 100% protection against severe/critical COVID-19 @14 days post-booster vaccination The …

Johnson & Johnson Reports EUA Submission to the US FDA for Supporting the Booster of its Single Shot COVID-19 Vaccine Candidate Read More »

AstraZeneca Seeks the US FDA’s EUA for AZD7442 to Treat COVID-19 in US

AstraZeneca, COVID-19, EUA, FDA, regulatory, US, ZD7442 / October 5, 2021 October 5, 2021

Shots: AstraZeneca has submitted a request to the US FDA seeking EUA for its AZD7442 to treatprophylaxis of symptomatic COVID-19. If EUA is granted, AZD7442 will be the first LAAB for COVID-19 prevention The filing is based on the safety and efficacy data from the P-III PROVENT & STORM CHASER trials along with the P-I …

AstraZeneca Seeks the US FDA’s EUA for AZD7442 to Treat COVID-19 in US Read More »

ViiV Healthcare Submits Regulatory Applications to the US FDA for a New Dispersible Tablet Formulation of Abacavir/Dolutegravir/Lamivudine to Treat HIV in Children

(dolutegravir), abacavir, approval, FDA, HIV, lamivudine, regulatory, Triumeq, US, ViiV Healthcare / October 4, 2021 October 4, 2021

Shots: ViiV Healthcare has reported a regulatory submission to the US FDA for the approval of a new dispersible tablet formulation of the fixed-dose combination of abacavir, dolutegravir & lamivudine. The submission is based on modeled data and in line with regulatory recommendations The company expands its approval for Triumeq (abacavir/ dolutegravir/ lamivudine) to lower …

ViiV Healthcare Submits Regulatory Applications to the US FDA for a New Dispersible Tablet Formulation of Abacavir/Dolutegravir/Lamivudine to Treat HIV in Children Read More »

Kite’s Tecartus Receives the US FDA’s Approval as the First and Only Car T for Adults with Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia

Acute Lymphoblastic Leukemia, approval, B-cell, FDA, Kite, Refractory, regulatory, Relapsed, Tecartus, US / October 4, 2021 October 4, 2021

Shots: The approval was based on a P-I/II ZUMA-3 study assessing Tecartus in adult patients aged ≥18yrs. with ALL whose disease is refractory to or has relapsed following first standard systemic therapy with remission of 12mos. or less, after 2L+ systemic therapy or at least 100 days after ASCT Results: 65% of the evaluable patients …

Kite’s Tecartus Receives the US FDA’s Approval as the First and Only Car T for Adults with Relapsed or Refractory B-cell Acute Lymphoblastic Leukemia Read More »

BMS Reports EMA’s Validation of MAA for Relatlimab and Nivolumab as 1L Treatment for Unresectable or Metastatic Melanoma

BMS, EMA, MAA, metastatic melanoma, Nivolumab, regulatory, Relatlimab / October 1, 2021 October 1, 2021

Shots: The MAA was based on the efficacy & safety results from the P-II/III RELATIVITY-047 trial that evaluates the fixed-dose combination of relatlimab (160mg) + nivolumab (480mg) vs Opdivo (480mg, IV, q4w) alone in a ratio (1:1) in 714 patients aged ≥12yrs. with previously untreated metastatic or unresectable melanoma The results demonstrated an improvement in …

BMS Reports EMA’s Validation of MAA for Relatlimab and Nivolumab as 1L Treatment for Unresectable or Metastatic Melanoma Read More »

TransMedics’ OCS Liver System Receives the US FDA’s Approval for Liver Transplantation

approval, FDA, Livers Transplantation, MedTech, OCS Liver System, regulatory, TransMedics, US / September 30, 2021 September 30, 2021

Shots: The US FDA has granted premarket approval of TransMedics’s OCS liver system for use with organs from DBD and DCD. The approval is based on the results of the OCS Liver PROTECT trial The OCS liver system is designed to preserve and monitor hemodynamics and metabolic function which allows ex-vivo assement of liver allografts …

TransMedics’ OCS Liver System Receives the US FDA’s Approval for Liver Transplantation Read More »

AbbVie’s Qulipta (atogepant) Receives the US FDA’s Approval for the Treatment of Migraine

AbbVie, approval, Atogepant, FDA, Migraine, Qulipta, regulatory, US / September 29, 2021 September 29, 2021

Shots: The approval was based on a clinical program including P-III ADVANCE study, P-IIb/III study & P-III LTS study evaluated the safety, efficacy & tolerability of Qulipta (PO, 10/30/60mg) vs PBO in 2000 patients with EM who experienced 4-14 migraine days/mos. The P-III ADVANCE & P-IIb/III study met its 1EPs i.e., reduction in mean monthly …

AbbVie’s Qulipta (atogepant) Receives the US FDA’s Approval for the Treatment of Migraine Read More »

The US FDA Approves New Indication for Lilly’s Erbitux (cetuximab) + Braftovi (encorafenib) to Treat Metastatic Colorectal Cancer with a BRAF V600E Mutation

approval, BRAF V600E Mutation, Braftovi, cetuximab, Encorafenib, Erbitux, FDA, Lilly, Metastatic Colorectal Cancer, regulatory, US / September 29, 2021 September 29, 2021

Shots: The approval is based on P-III BEACON CRC trial evaluating Braftovi (300mg, qd) + Erbitux vs irinotecan + Erbitux or Folfiri + Erbitux in a ratio (1:1:1) in 441 patients with previously treated mCRC with a BRAF V600E mutation after prior therapy Erbitux + Braftovi showed an improved OS; m-OS (8.4 vs 5.4mos.); ORR …

The US FDA Approves New Indication for Lilly’s Erbitux (cetuximab) + Braftovi (encorafenib) to Treat Metastatic Colorectal Cancer with a BRAF V600E Mutation Read More »

Eisai Initiates Rolling Submission of BLA to the US FDA for Lecanemab to Treat Alzheimer’s Disease

BLA, Eisai, FDA, lecanemab, regulatory, US / September 28, 2021 September 28, 2021

Shots: The submission is based on clinical, biomarker & safety data of Study 201 POC study evaluates lecanemab (10mg/kg, q2w) in 856 patients with MCI due to AD with confirmed presence of amyloid pathology. The BLA is being submitted under accelerated approval The results showed a reduction in brain amyloid by 0.306 SUVr units @18mos. …

Eisai Initiates Rolling Submission of BLA to the US FDA for Lecanemab to Treat Alzheimer’s Disease Read More »

AstraZeneca’s Saphnelo (anifrolumab) Receives MHLW’s Approval for the Treatment of Systemic Lupus Erythematosus

anifrolumab, approval, AstraZeneca, MHLW, regulatory, Saphnelo, Systemic Lupus Erythematosus / September 28, 2021 September 28, 2021

Shots: The approval was based on the clinical program including two P-III TULIP & P-II MUSE trials evaluated the efficacy and safety of Saphnelo vs PBO in 362 & 305 patients with SLE who are receiving standard therapy The results showed a reduction in overall disease activity across organ systems, including skin and joints, and …

AstraZeneca’s Saphnelo (anifrolumab) Receives MHLW’s Approval for the Treatment of Systemic Lupus Erythematosus Read More »

Takeda’s Alofisel (darvadstrocel) Receives MHLW’s Approval for the Treatment of Complex Perianal Fistulas with Non-active or Mildly Active Luminal CD

regulatory / September 27, 2021 September 27, 2021

Shots: The approval is based on the two trials i.e., P-III Darvadstrocel-3002 & ADMIRE-CD trial evaluating the safety & efficacy of Alofisel in 22 & 212 patients with CPF with non-active/mildly active luminal CD The results from the P-III ADMIRE-CD trial showed that the proportion of patients in Alofisel over the control group achieved 1EPs …

Takeda’s Alofisel (darvadstrocel) Receives MHLW’s Approval for the Treatment of Complex Perianal Fistulas with Non-active or Mildly Active Luminal CD Read More »

Astellas and Seagen’s Padcev (enfortumab vedotin) Receive MHLW’s Approval for the Treatment of Radically Unresectable Urothelial Carcinoma

regulatory / September 27, 2021 September 27, 2021

Shots: The approval is based on the P-III EV-301 clinical trial evaluating Padcev vs CT in 608 patients with LA/mUC prior treated with Pt-based CT and a PD-1/L1 inhibitor The pre-specified interim analysis showed that patients treated with Padcev demonstrated an m-OS (12.9 vs 9.0mos.) with a median of 3.9mos. longer than those who received …

Astellas and Seagen’s Padcev (enfortumab vedotin) Receive MHLW’s Approval for the Treatment of Radically Unresectable Urothelial Carcinoma Read More »

Amgen’s Repatha (evolocumab) Receives the US FDA’s Approval for the Treatment of Heterozygous Familial Hypercholesterolemia

Amgen, approval, evolocumab, FDA, Heterozygous Familial Hypercholesterolemia, regulatory, Repatha, US / September 27, 2021 September 27, 2021

Shots: The approval is based on the P-IIIb HAUSER-RCT study evaluating the safety & efficacy of Repatha (420mg, SC) vs PBO in a ratio (2:1) in pediatric patients aged 10 – 17yrs. with HeFH for 24wks. The trial met its 1EPs i.e., 38% reduction in LDL-C from baseline, patients treated with Repatha had improved secondary …

Amgen’s Repatha (evolocumab) Receives the US FDA’s Approval for the Treatment of Heterozygous Familial Hypercholesterolemia Read More »

MedCity Pivot Podcast: A startup co-founder on the urgent changes needed in women’s healthcare

Carolyn Witte, CommonSpirit Health, Company, Daily, health inequities, Health IT, income, News, race, regulatory, Startups, SYN, Tia, Top Story, womens health / September 26, 2021 September 26, 2021

The siloed approach to women’s healthcare is one of the main reasons the U.S. is unable to improve its record in this arena, according to Carolyn Witte, co-founder of startup Tia. In our latest episode, she discusses how her company is trying to solve this issue while also working to close the gaps that deepen …

MedCity Pivot Podcast: A startup co-founder on the urgent changes needed in women’s healthcare Read More »

Everest’s SPR206 Receives the NMPA’s IND Approval for the Treatment of MDR Gram-Negative Bacterial Infections

approval, Everest, IND, MDR Gram-Negative Bacterial Infections, NMPA, regulatory, SPR206 / September 24, 2021 September 24, 2021

Shots: The NMPA has approved the IND application of SPR206 (IV) for the treatment of MDR gram-negative bacterial infections. Everest will initiate the clinical program in China & results from the P-I study is expected in 2022 Everest got exclusive rights to develop, manufacture & commercialize SPR206 in China, South Korea, and Southeast Asian countries …

Everest’s SPR206 Receives the NMPA’s IND Approval for the Treatment of MDR Gram-Negative Bacterial Infections Read More »

Pfizer and BioNTech’s COVID-19 Vaccine Booster Receive the US FDA’s EUA for the Treatment of COVID-19

BioNTech, COVID-19, COVID-19 Vaccine Booster, EUA, FDA, Pfizer, regulatory, US / September 24, 2021 September 24, 2021

Shots: The US FDA has issued a EUA for the COVID-19 booster vaccine (administered at least 6mos. after completion of primary 2 doses) to prevent COVID-19 in individuals aged ≥65yrs. & 18 through 64yrs. of age who at high risk of severe COVID-19 The EUA is based on clinical data that showed higher neutralizing Ab …

Pfizer and BioNTech’s COVID-19 Vaccine Booster Receive the US FDA’s EUA for the Treatment of COVID-19 Read More »

Incyte’s Jakafi (ruxolitinib) Receives the US FDA’s Approval for the Treatment of Chronic Graft-Versus-Host Disease

approval, Chronic Graft Versus Host Disease, FDA, Incyte, Jakafi, regulatory, ruxolitinib, US / September 23, 2021 September 23, 2021

Shots: The approval is based on the P-III REACH3 study evaluates the safety & efficacy of Jakafi vs BAT in patients ≥12yrs. with steroid-refractory chronic GVHD after the failure of 1 or 2 lines of systemic therapy The results showed that the patients treated with Jakafi led to significantly improved outcomes across a range of …

Incyte’s Jakafi (ruxolitinib) Receives the US FDA’s Approval for the Treatment of Chronic Graft-Versus-Host Disease Read More »

Innovent Reports NMPA’s Acceptance of sNDA for Sintilimab as 1L Treatment for Esophageal Squamous Cell Carcinoma

regulatory / September 23, 2021 September 23, 2021

Shots: The sNDA is based on the P-III ORIENT-15 clinical trial evaluates sintilimab + CT vs PBO + CT in a ratio (1:1) in 659 patients unresectable LA, recurrent or metastatic ESCC In an interim analysis, the therapy demonstrated an improvement in the 1EPs of OS & PFS. The results were presented at ESMO 2021 …

Innovent Reports NMPA’s Acceptance of sNDA for Sintilimab as 1L Treatment for Esophageal Squamous Cell Carcinoma Read More »

Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv) Receive the US FDA’s Accelerated Approval for the Treatment of Recurrent or Metastatic Cervical Cancer

Accelerated Approval, cervical cancer, FDA, Genmab, regulatory, Seagen, tisotumab vedotin-tftv, Tivdak, US / September 22, 2021 September 22, 2021

Shots: The approval is based on the P-II innovaTV 204 clinical trials evaluating tisotumab vedotin in 101 patients with r/mCC who had received no more than two prior systemic regimens in the recurrent or metastatic setting, including one prior Pt-based CT regimen The results showed an ORR (24%) as assessed by IRC using RECIST v1.1 …

Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv) Receive the US FDA’s Accelerated Approval for the Treatment of Recurrent or Metastatic Cervical Cancer Read More »

Incyte’s Opzelura (ruxolitinib) Receives the US FDA’s Approval for the Treatment of Atopic Dermatitis

approval, Atopic Dermatitis, FDA, Incyte, Opzelura, regulatory, ruxolitinib, US / September 22, 2021 September 22, 2021

Shots: The approval is based on the TRuE-AD clinical trial program which consists of two P-III studies i.e., TRuE-AD1 & 2 evaluating the safety and efficacy of Opzelura cream (1.5%, bid) vs vehicle (non-medicated cream) in 1200+ adolescents & adults aged ≥12yrs. with mild to moderate AD The results showed that the patients achieved IGA-TS …

Incyte’s Opzelura (ruxolitinib) Receives the US FDA’s Approval for the Treatment of Atopic Dermatitis Read More »

Samsung Bioepis and Biogen’s Byooviz (biosimilar, ranibizumab) Receives the US FDA’s Approval for the Treatment of Retinal Vascular Disorders

approval, Biogen, Biosimilar, Biosimilars, Byooviz, FDA, ranibizumab, regulatory, Retinal Vascular Disorders, Samsung Bioepis, US / September 21, 2021 September 21, 2021

Shots: The approval is based on a totality of evidence including analytical, non-clinical/clinical data from the P-III study evaluates the efficacy, safety, PK & immunogenicity of SB11 (0.5mg) vs Lucentis in a ratio (1:1) in 705 patients with wet AMD The results showed LS mean change in BCVA from baseline @52wks. (9.79 vs 10.41 letters) …

Samsung Bioepis and Biogen’s Byooviz (biosimilar, ranibizumab) Receives the US FDA’s Approval for the Treatment of Retinal Vascular Disorders Read More »

Exelixis’ Cabometyx (cabozantinib) Receives the US FDA’s Approval for the Treatment of Locally Advanced or Metastatic Differentiated Thyroid Cancer

(cabozantinib), approval, Cabometyx, Differentiated Thyroid Cancer, Exelixis, FDA, regulatory, US / September 20, 2021 September 20, 2021

Shots: The approval is based on P-III COSMIC-311 evaluating Cabometyx (60mg, qd) vs PBO in a ratio (2:1) in 258 patients aged ≥12yrs. with radioactive iodine-refractory DTC who progressed after up to two prior VEGFR-targeted therapies across 164 sites globally At a planned interim analysis, the therapy demonstrated the reduction in the risk of disease …

Exelixis’ Cabometyx (cabozantinib) Receives the US FDA’s Approval for the Treatment of Locally Advanced or Metastatic Differentiated Thyroid Cancer Read More »

AbbVie Submits Regulatory Applications to the US FDA and EMA for Rinvoq (upadacitinib) to Treat Active Ulcerative Colitis

regulatory / September 17, 2021 September 17, 2021

Shots: The application is based on 2 P-III U-ACHIEVE & U-ACCOMPLISH induction studies & 1 U-ACHIEVE maintenance study evaluating upadacitinib (45mg, qd) as IT & (15/30 mg, qd) as MT vs PBO in patients with mod. to sev. UC The studies met its 1EPs i.e., patients achieved clinical remission (per Adapted Mayo Score) & 2EPs …

AbbVie Submits Regulatory Applications to the US FDA and EMA for Rinvoq (upadacitinib) to Treat Active Ulcerative Colitis Read More »

BioCryst’s Orladeyo (berotralstat) Receives NICE Recommendation as First Oral Therapy to Prevent HAE Attacks in the UK

regulatory / September 16, 2021 September 16, 2021

Shots: The NICE recommendation is based on P-III APeX-2 trial evaluating Orladeyo vs PBO in adult & pediatric patients aged ≥12yrs. with HAE attacks The trial met its 1EPs i.e., reduction in HAE attacks @24wks. & sustained through 96wks. with an 80% reduction in patients with mean HAE attack rate/mos. @ 25-96wks. The therapy was …

BioCryst’s Orladeyo (berotralstat) Receives NICE Recommendation as First Oral Therapy to Prevent HAE Attacks in the UK Read More »

BeiGene’s Brukinsa (zanubrutinib) Receives the US FDA’s Accelerated Approval for the Treatment of R/R Marginal Zone Lymphoma

Accelerated Approval, BeiGene, Brukinsa, FDA, R/R Marginal Zone Lymphoma, regulatory, US, Zanubrutinib / September 15, 2021 September 15, 2021

Shots: The approval is based on 2 trials i.e., P-II MAGNOLIA & P-I/II BGB-3111-AU-003 trial evaluate Brukinsa (160mg, bid or 320 mg, qd) in 66 & 20 patients with R/R MZL who received at least 1 anti-CD20-based regimen Both trial demonstrated ORR (56% & 80%), CR rate (20% & 20%) based on assessment using CT …

BeiGene’s Brukinsa (zanubrutinib) Receives the US FDA’s Accelerated Approval for the Treatment of R/R Marginal Zone Lymphoma Read More »

Amgen’s Lumakras (sotorasib) Receives Health Canada’s Approval for KRAS G12C-Mutated Locally Advanced or Metastatic Non-Small Cell Lung Cancer

Amgen, approval, Health Canada, KRAS G12C-Mutated, Lumakras, Non-Small Cell Lung Cancer, regulatory, Sotorasib / September 14, 2021 September 14, 2021

Shots: The approval is based on P-II CodeBreaK 100 trial evaluating the efficacy and tolerability of Lumakras (PO, qd) in 126 patients with KRAS G12C mutation-positive advanced NSCLC whose disease had progressed after prior treatment with immunotherapy or CT The 1EPs were centrally assessed ORR. Additionally, the therapy demonstrated a positive benefit-risk profile with rapid, …

Amgen’s Lumakras (sotorasib) Receives Health Canada’s Approval for KRAS G12C-Mutated Locally Advanced or Metastatic Non-Small Cell Lung Cancer Read More »

Hutchmed’s Amdizalisib (HMPL-689) Receives Breakthrough Therapy Designation for the Treatment of R/R Follicular Lymphoma in China

regulatory / September 13, 2021 September 13, 2021

Shots: The NMPA has granted BTD to amdizalisib for the treatment of r/r FL The preliminary results from the ongoing P-Ib expansion study showed that the therapy was well- tolerated, exhibit dose-proportional PK, manageable toxicity profile, clinical activity in r/r B-cell lymphoma & results will be presented at ESMO 2021. Currently, the company gains all …

Hutchmed’s Amdizalisib (HMPL-689) Receives Breakthrough Therapy Designation for the Treatment of R/R Follicular Lymphoma in China Read More »

BeiGene and Novartis Report the US FDA’s Acceptance of BLA for Tislelizumab to Treat Esophageal Squamous Cell Carcinoma

BeiGene, BLA, Esophageal Squamous Cell Carcinoma, FDA, regulatory, Tislelizumab, US / September 13, 2021 September 13, 2021

Shots: The BLA submission is based on a P-III RATIONALE 302 trial evaluating the efficacy and safety of tislelizumab vs CT in 1972 patients with advanced or metastatic ESCC who had received prior systemic therapy. The anticipated PDUFA date for the therapy is July 12, 2022 The trial met its 1EPs i.e., the therapy showed …

BeiGene and Novartis Report the US FDA’s Acceptance of BLA for Tislelizumab to Treat Esophageal Squamous Cell Carcinoma Read More »

Boehringer Ingelheim and Eli Lilly’s Jardiance (empagliflozin) Receive the US FDA’s Breakthrough Therapy Designation for the Treatment of Heart Failure with Preserved Ejection Fraction

Boehringer Ingelheim, Breakthrough Therapy Designation, Eli Lilly, Empagliflozin, heart failure, Jardiance, Preserved Ejection Fraction, regulatory, US / September 10, 2021 September 10, 2021

Shots: The designation is based on the P-III EMPEROR-Preserved trial evaluates the safety and efficacy of empagliflozin (10mg, qd) vs PBO in 5,988 patients with chronic HFpEF with/out diabetes The results demonstrated a 21% reduction in risk for the composite 1EPs of CV death or hospitalization for adults with HF. The results were presented at …

Boehringer Ingelheim and Eli Lilly’s Jardiance (empagliflozin) Receive the US FDA’s Breakthrough Therapy Designation for the Treatment of Heart Failure with Preserved Ejection Fraction Read More »

MHRA cuts could affect UK regulatory decisions, say unions

Brexit, Market Access, MHRA, News, regulatory, Workforce / September 8, 2021 September 8, 2021

A 20% to 25% reduction in staffing at the UK Medicines and Healthcare products Regulatory Agency (MHRA) risks undermining the ability of the authority to fulfil its role, according to trade unions. In an open letter to Health Secretary Sajid Javid, Prospect, Unite and three other unions say the decision to slash the MHRA’s current …

MHRA cuts could affect UK regulatory decisions, say unions Read More »

Impel NeuroPharma’s Trudhesa (dihydroergotamine mesylate) Nasal Spray Receives the US FDA’s Approval for the Treatment of Migraine

approval, dihydroergotamine mesylate, FDA, impel neuropharma, Migraine, nasal spray, regulatory, Trudhesa, US / September 7, 2021 September 7, 2021

Shots: The approval is based on the P-III STOP 301 trial evaluates Trudhesa (0.725 mg/spray) in 5650+ patients with migraine with or without aura for 24 or 52 wks. The exploratory efficacy results showed that nasal spray provides rapid, sustained, and consistent symptom relief. The therapy is well-tolerated & no serious TEAEs were observed Trudhesa …

Impel NeuroPharma’s Trudhesa (dihydroergotamine mesylate) Nasal Spray Receives the US FDA’s Approval for the Treatment of Migraine Read More »

JW’s Relmacabtagene Autoleucel Receives the NMPA’s Approval for the Treatment of R/R Large B-Cell Lymphoma in China

approval, China, JW, NMPA, R/R Large-B-Cell Lymphoma, regulatory, Relmacabtagene Autoleucel / September 7, 2021 September 7, 2021

Shots: The approval is based on a RELIANCE study that evaluates efficacy & safety of relma-cel in 59 patients with r/r LBCL who had failed at least 2L of therapy, including CD20 agent & anthracycline & patients continue to be monitored for 2+ yrs. The results showed the high rates of durable disease response & …

JW’s Relmacabtagene Autoleucel Receives the NMPA’s Approval for the Treatment of R/R Large B-Cell Lymphoma in China Read More »

Moderna’s Spikevax Receives TGA’s Provisional Approval to Treat COVID-19 in Patients Aged 12-17 Years

Australia, COVID-19, Moderna, regulatory, Spikevax, TGA / September 6, 2021 September 6, 2021

Shots: TGA has provisionally approved the use of Moderna’s Spikevax (elasomeran) in individuals aged ≥12yrs. The decision follows the provisional approval granted by the TGA to Spikevax on Aug 09, 2021 for use in individuals aged ≥18yrs. Provisional approval for this group follows the data supporting the efficacy & safety of the vaccine. The recommended …

Moderna’s Spikevax Receives TGA’s Provisional Approval to Treat COVID-19 in Patients Aged 12-17 Years Read More »

Novartis Cosentyx (secukinumab) Receives NICE Recommendation for Patients with Plaque Psoriasis

Cosentyx, NICE Recommendation, Novartis, regulatory, secukinumab / September 3, 2021 September 3, 2021

Shots: NICE has recommended Cosentyx as a cost-effective option for the treatment of mod. to sev. PsO in patients aged 6-17 yrs. who have not responded to other forms of systemic treatment & for whom these options were contraindicated or not tolerated Additionally, patients will now have access to this treatment option on the NHS. …

Novartis Cosentyx (secukinumab) Receives NICE Recommendation for Patients with Plaque Psoriasis Read More »

Alexion’s Ultomiris (ravulizumab) Receives EC’s Approval for Expanded Use to Treat Paroxysmal Nocturnal Haemoglobinuria

Alexion, approval, EC, EU, Paroxysmal Nocturnal Haemoglobinuria, ravulizumab, regulatory, Ultomiris / September 3, 2021 September 3, 2021

Shots: The approval is based on the P-III trial that evaluates Ultomiris (q4w/q8w) vs Soliris in children & adolescents aged ≤18yrs. with PNH The interim result showed that the therapy was effective in achieving C5 complement inhibition @26wks. & demonstrated safety & efficacy profile with reduced dosing frequency, no treatment-related SAEs & no patients discontinued …

Alexion’s Ultomiris (ravulizumab) Receives EC’s Approval for Expanded Use to Treat Paroxysmal Nocturnal Haemoglobinuria Read More »

Janssen’s Invega Hafyera (paliperidone palmitate) Receives the US FDA’s Approval for the Treatment of Schizophrenia

approval, FDA, Invega Hafyera, Janssen, paliperidone palmitate, regulatory, Schizophrenia, US / September 2, 2021 September 2, 2021

Shots: The approval is based on a P-III study that evaluates Invega hafyera (q6mos., twice-yearly injectable) vs Invega trinza in 702 adults in a ratio (2:1) aged 18-70yrs. with schizophrenia from 20 countries The results showed non-inferiority on 1EPs of time to 1st relapse @12mos. in ITT & per-protocol analysis sets., patients were relapse-free (92.5% …

Janssen’s Invega Hafyera (paliperidone palmitate) Receives the US FDA’s Approval for the Treatment of Schizophrenia Read More »

BeiGene’s Brukinsa (zanubrutinib) Receives the US FDA’s Approval for the Treatment of Waldenström’s Macroglobulinemia

approval, BeiGene, Brukinsa, FDA, regulatory, US, Waldenström’s Macroglobulinemia, Zanubrutinib / September 2, 2021 September 2, 2021

Shots: The approval is based on P-III ASPEN trial evaluating Brukinsa (160mg, bid, or 320 mg, qd) vs ibrutinib in 201 patients with WM who harbor an MYD88 mutation The 1EPs of the trial was VGPR rate in the overall ITT population as assessed by IRC. VGPR rate (28% vs 19%) based on the modified …

BeiGene’s Brukinsa (zanubrutinib) Receives the US FDA’s Approval for the Treatment of Waldenström’s Macroglobulinemia Read More »

Merck’s Keytruda (pembrolizumab) Receives the US FDA’s Approval for Label Update to Treat Advanced Urothelial Carcinoma

regulatory / September 1, 2021 September 1, 2021

Shots: The US FDA has approved Merck’s Keytruda for the treatment of patients with locally advanced or mUC who are not eligible for any Pt-containing CT & converted the indication from an accelerated to a full approval along with a label update Previously, the FDA’s ODAC voted 5-3 in favor of maintaining the accelerated approval …

Merck’s Keytruda (pembrolizumab) Receives the US FDA’s Approval for Label Update to Treat Advanced Urothelial Carcinoma Read More »

Ascendis’ Skytrofa (lonapegsomatropin-tcgd) Receives the US FDA’s Approval for Pediatric Growth Hormone Deficiency

approval, Ascendis, FDA, lonapegsomatropin-tcgd, Pediatric Growth Hormone Deficiency, regulatory, Skytrofa, US / August 27, 2021 August 27, 2021

Shots: The approval is based on the P-III heiGHt trial evaluating Skytrofa (qw) vs somatropin in 161 treatment-naïve children ≥aged 1yrs. with GHD who have growth failure due to inadequate secretion of endogenous GH The study met its 1EPs i.e., non-inferiority in AHV & showed a higher AHV @52wks., no serious AEs or discontinuations related …

Ascendis’ Skytrofa (lonapegsomatropin-tcgd) Receives the US FDA’s Approval for Pediatric Growth Hormone Deficiency Read More »

MorphoSys and Incyte’s Minjuvi (tafasitamab) + Lenalidomide Receive EC’s Approval for the Treatment of R/R DLBCL

approval, EC, Incyte, Lenalidomide, Minjuvi, MorphoSys, regulatory, tafasitamab / August 27, 2021 August 27, 2021

Shots: The conditional approval is based on the P-II L-MIND study evaluating the safety and efficacy of tafasitamab + lenalidomide in patients with r/r DLBCL who are not eligible for ASCT Results: ORR (56.8%); CR rate (39.5%); PR rate (17.3%) as assessed by an IRC; m-DoR (43.9mos.) after a minimum follow up of 35mos. Tafasitamab …

MorphoSys and Incyte’s Minjuvi (tafasitamab) + Lenalidomide Receive EC’s Approval for the Treatment of R/R DLBCL Read More »

Pfizer and BioNTech Initiate Rolling Submission of sBLA for the US FDA’s Approval of Comirnaty’s Booster Dose in Individuals Aged 16 Years and Older

regulatory / August 26, 2021 August 26, 2021

Shots: The sBLA is based on P-III trial for Comirnaty (third booster dose) administered b/w 4.8 – 8mos., following the completion of 2 dose in 306 participants aged b/w 18-55 yrs. The P-III trial showed that third booster dose induces robust neutralizing Abs against wild-type strain @1mos., following PD3 with a median follow-up time of …

Pfizer and BioNTech Initiate Rolling Submission of sBLA for the US FDA’s Approval of Comirnaty’s Booster Dose in Individuals Aged 16 Years and Older Read More »

AstraZeneca’s Forxiga (dapagliflozin) Receives MHLW’s Approval for the Treatment of Chronic Kidney Disease in Patients with and without T2D

regulatory / August 26, 2021 August 26, 2021

Shots: The approval is based on P-III DAPA-CKD trial evaluate Forxiga (10mg, qd) + SoC vs PBO in 4304 patients with CKD Stage 2-4 & elevated urinary albumin excretion with/out T2D The results showed a 39% reduction in risk of worsening of renal function, ESKD, CV, or renal death & 31% from any cause. Safety …

AstraZeneca’s Forxiga (dapagliflozin) Receives MHLW’s Approval for the Treatment of Chronic Kidney Disease in Patients with and without T2D Read More »

Thermo Fisher’s Oncomine Dx Target Test Receives the US FDA’s Approval as a CDx for the Treatment of IDH1-Mutated Cholangiocarcinoma

approval, CDx, FDA, IDH1-Mutated Cholangiocarcinoma, MedTech, Oncomine Dx Target Test, regulatory, Thermo Fisher, US / August 26, 2021 August 26, 2021

Shots: The FDA has approved the Oncomine Dx target test as a CDx to identify patients with IDH1 mutated CCA who may be candidates for Servier’s Tibsovo Oncomine Dx Target Test is NGS based CDx test that provides robust results in the IDH1 gene clinically associated with CCA & is currently approved in 15+ countries, …

Thermo Fisher’s Oncomine Dx Target Test Receives the US FDA’s Approval as a CDx for the Treatment of IDH1-Mutated Cholangiocarcinoma Read More »

Cara and Vifor’s Korsuva (difelikefalin) Receive the US FDA’s Approval for the Treatment of Moderate-to-Severe Pruritus Due to CKD in Patients with Hemodialysis

regulatory / August 25, 2021 August 25, 2021

Shots: The approval is based on the two P-III trials i.e., KALM-1, KALM-2 along with additional 32 clinical studies that evaluate Korsuva vs PBO in patients with a mod. to sev. pruritus associated with CKD who undergoes hemodialysis Results: patients achieved four-point improvement from baseline on a measure of severe itch (40% & 37%) vs …

Cara and Vifor’s Korsuva (difelikefalin) Receive the US FDA’s Approval for the Treatment of Moderate-to-Severe Pruritus Due to CKD in Patients with Hemodialysis Read More »

Argenx Reports EMA’s Validation of MAA for Efgartigimod to Treat Generalized Myasthenia Gravis

Argenx, Efgartigimod, EMA, MAA, regulatory / August 25, 2021 August 25, 2021

Shots: The MAA is based on the P-III ADAPT trial evaluating the safety and efficacy of efgartigimod vs PBO in a ratio (1:1) in 167 adult patients with gMG for 26wks. The 1EPs was the proportion of AChR-Ab+ patients who achieved a response on the MG-ADL score defined by at least a two-point improvement for …

Argenx Reports EMA’s Validation of MAA for Efgartigimod to Treat Generalized Myasthenia Gravis Read More »

Takeda and Ono’s Opdivo (nivolumab) + Cabometyx (cabozantinib) Receive MHLW’s Approval for the Treatment of Unresectable or Metastatic Renal Cell Carcinoma

regulatory / August 25, 2021 August 25, 2021

Shots: The approval is based on P-III CheckMate -9ER study evaluating ONO’s Opdivo (240 mg, IV, q2w) + Takeda’s Cabometyx (40mg, PO, qd) vs sunitinib (50mg, PO, qd, for 4wks.) in a ratio (1:1) in patients with previously untreated advanced/mRCC Opdivo + Cabometyx demonstrated an improvement in 1EPs of PFS & 2EPs of OS & …

Takeda and Ono’s Opdivo (nivolumab) + Cabometyx (cabozantinib) Receive MHLW’s Approval for the Treatment of Unresectable or Metastatic Renal Cell Carcinoma Read More »

AbbVie’s Rinvoq (upadacitinib) Receives EC’s Approval for the Treatment of Moderate to Severe Atopic Dermatitis

AbbVie, approval, EC, for the Treatment of Moderate to Severe Atopic Dermatitis, regulatory, Rinvoq, Upadacitinib / August 25, 2021 August 25, 2021

Shots: The approval is based on 3 P-III studies i.e., Measure Up 1/2/AD Up evaluating the efficacy & safety of Rinvoq (15/30 mg, qd) as monothx. with TCS vs PBO in 2500+ adults & adolescents with AD The studies met its 1EPs & 2EPs @16wks. i.e., improvement in skin clearance & itch reduction, patients achieved …

AbbVie’s Rinvoq (upadacitinib) Receives EC’s Approval for the Treatment of Moderate to Severe Atopic Dermatitis Read More »

UCB’s Bimzelx (bimekizumab) Receives EC’s Approval for the Treatment of Moderate to Severe Plaque Psoriasis

approval, bimekizumab, Bimzelx, EC, Plaque Psoriasis, regulatory, UCB / August 24, 2021 August 24, 2021

Shots: The approval is based on 3 P-III studies i.e., BE VIVID, BE READY, BE SURE evaluating the efficacy & safety of bimekizumab vs PBO + ustekinumab, PBO, adalimumab in 1480 patients with a mod. to sev. PsO. The EC’s approval is valid in 27 member states of the EU, Iceland, Liechtenstein & Norway The …

UCB’s Bimzelx (bimekizumab) Receives EC’s Approval for the Treatment of Moderate to Severe Plaque Psoriasis Read More »

Pfizer and BioNTech’s Comirnaty Receive the US FDA’s Approval to Prevent COVID-19 in Individuals Aged 16 Years and Older

approval, Comirnaty, COVID-19, Covid-19 Vaccine, FDA, Pfizer/BioNTech, regulatory, US / August 24, 2021 August 24, 2021

Shots: The approval is based on the longer-term follow-up data from the P-III trial that showed high efficacy and favorable safety profile through 6mos. after the second dose The vaccine will now be marketed as Comirnaty and is fully approved for the prevention of COVID-19 in individuals aged ≥16 yrs. The companies also plan to …

Pfizer and BioNTech’s Comirnaty Receive the US FDA’s Approval to Prevent COVID-19 in Individuals Aged 16 Years and Older Read More »

Zydus’ ZyCoV-D Receives the DCGI’s EUA for the Treatment of COVID-19

COVID-19, DCGI, EUA, regulatory, ZyCoV-D, Zydus / August 23, 2021 August 23, 2021

Shots: The DCGI has granted a EUA for ZyCoV-D which is the world’s first plasmid DNA vaccine for COVID-19. The company is planning to seek approval for the two-dose regimen of the vaccine ZyCoV-D will become India’s first vaccine for adolescents aged 12-18 yrs. The vaccine is safe and well-tolerated ZyCoV-D is a three-dose, needle-free, …

Zydus’ ZyCoV-D Receives the DCGI’s EUA for the Treatment of COVID-19 Read More »

Pfizer’s Xeljanz (tofacitinib citrate) Receives EC’s Approval for the Treatment of Active Polyarticular JIA & Juvenile PsA

Active Polyarticular JIA, EC Approval, Juvenile PsA, Pfizer, regulatory, tofacitinib citrate, XELJANZ / August 23, 2021 August 23, 2021

Shots: The approval is based on a P-III study which includes 2 phases i.e., run-in & withdrawal phase evaluating efficacy & safety of tofacitinib (5mg tablet or mg/mL oral solution, BID) in 398 patients aged ≥2yrs. with polyarticular JIA & juvenile PsA who have had an inadequate response to previous therapy with DMARDs for 44wks. …

Pfizer’s Xeljanz (tofacitinib citrate) Receives EC’s Approval for the Treatment of Active Polyarticular JIA & Juvenile PsA Read More »

BeiGene Reports NMPA’s Acceptance of sBLA for Tislelizumab (BGB-A317) to Treat Nasopharyngeal Cancer in China

BeiGene, China, Nasopharyngeal Cancer, NMPA, regulatory, sBLA, Tislelizumab / August 23, 2021 August 23, 2021

Shots: The sBLA is based on P-III RATIONALE 309 trial evaluating the efficacy & safety of tislelizumab + CT vs PBO + CT in a ratio (1:1) in 263 Asian patients with recurrent or metastatic NPC The 1EPs of a trial is PFS as assessed by IRC in ITT population & 2EPs includes OS, ORR, …

BeiGene Reports NMPA’s Acceptance of sBLA for Tislelizumab (BGB-A317) to Treat Nasopharyngeal Cancer in China Read More »

BMS’ Opdivo(nivolumab) Receives the US FDA’s Approval for the Adjuvant Treatment of High-Risk Urothelial Carcinoma

regulatory / August 23, 2021 August 23, 2021

Shots: The approval is based on the P-III CheckMate -274 trial evaluating Opdivo (240mg, IV, q2w, for ~1 yrs.) vs PBO in a ratio (1:1) in 709 patients with UC who are at high risk of recurrence after undergoing radical resection The results showed a 30% reduction in the risk of disease recurrence or death, …

BMS’ Opdivo(nivolumab) Receives the US FDA’s Approval for the Adjuvant Treatment of High-Risk Urothelial Carcinoma Read More »

Roche’s Ventana MMR RxDx Panel Test Receives the US FDA’s Approval as First CDx to Detect dMMR Solid Tumor

approval, dMMR Solid Tumor, FDA, MedTech, regulatory, Roche, US, Ventana MMR RxDx Panel Test / August 20, 2021 August 20, 2021

Shots: The FDA has approved the Ventana MMR RxDx Panel test as a CDx to identify patients with dMMR advanced solid tumors, who may be eligible for Jemperli monothx., based on the results of the MMR biomarker test Ventana MMR RxDx Panel provides access to clinicians with fully automated panel of MMR biomarkers tested by …

Roche’s Ventana MMR RxDx Panel Test Receives the US FDA’s Approval as First CDx to Detect dMMR Solid Tumor Read More »

Astellas and FibroGen’s Evrenzo (roxadustat) Receive EC’s Approval for Symptomatic Anemia Associated with Chronic Kidney Disease

Chile & South Korea for the same indication in adult patients on DD & NDD, China, including the EEA countries, Japan, regulatory, Roxadustat is a HIF-PH inhibitor & is approved in EU member states / August 20, 2021 August 20, 2021

Shots: The approval is based on the P-III program including 8 studies evaluating Evrenzo in 9600 patients with symptomatic anemia associated with CKD, regardless of dialysis status & prior ESA treatment. Following the EC’s approval, FibroGen to receive $120M milestones along with royalties based upon EU sales The results demonstrated that the therapy was effective …

Astellas and FibroGen’s Evrenzo (roxadustat) Receive EC’s Approval for Symptomatic Anemia Associated with Chronic Kidney Disease Read More »

Gilead Reports EMA’s Validation of MAA for Lenacapavir to Treat HIV-1 in People with Limited Therapy Options

EMA, Gilead, HIV-1, lenacapavir, MAA, regulatory, validation / August 20, 2021 August 20, 2021

Shots: The MAA is based on the ongoing P-II/III CAPELLA study evaluates the antiviral activity of lenacapavir (PO, SC, every 6mos.) in combination with other antiretroviral agents vs PBO in a ratio (2:1) in 36 patients with multi-drug resistant HIV-1 infection Results: viral load reduction from baseline of at least 0.5 log10 copies/mL for 14-day …

Gilead Reports EMA’s Validation of MAA for Lenacapavir to Treat HIV-1 in People with Limited Therapy Options Read More »

BMS’ Abecma (Idecabtagene Vicleucel) Receives EC’s Conditional Approval for Relapsed and Refractory Multiple Myeloma

Abecma, BMS, Conditional Approval, EC, idecabtagene vicleucel, multiple myeloma, regulatory / August 20, 2021 August 20, 2021

Shots: The conditional approval is based on KarMMa study evaluating Abecma (single infusion with a target dose of 420 x 106 CAR T cells) in 128 patients with r/r MM prior treated with 3L therapies including an immunomodulatory agent, a proteasome inhibitor & an anti-CD38 Ab and who are refractory to their last treatment Results: …

BMS’ Abecma (Idecabtagene Vicleucel) Receives EC’s Conditional Approval for Relapsed and Refractory Multiple Myeloma Read More »

Does draft RCT guide set the scene for future trials?

clinical research, Clinical Trials, patients, R&D, RCT, regulatory, Views & Analysis, Views and analysis / August 19, 2021 August 19, 2021

A common set of standards for randomised clinical trials (RCTs) aims to help researchers develop the drugs and interventions of the future – no matter what they do or where they are based. The Good Clinical Trials Collaborative (GCTC) has published a draft guidance document, and is asking for the life sciences sector to make …

Does draft RCT guide set the scene for future trials? Read More »

Boehringer Ingelheim and Eli Lilly’s Jardiance (empagliflozin) Receive the US FDA’s Approval for the Treatment of Heart Failure with Reduced Ejection Fraction

approval, Boehringer Ingelheim, Eli Lilly, Empagliflozin, FDA, Heart Failure with Reduced Ejection Fraction, Jardiance, regulatory, US / August 19, 2021 August 19, 2021

Shots: The approval is based on the P-III EMPEROR-Reduced trial evaluating the effect of Jardiance (10 mg, qd) vs PBO in 3730 adults with HFrEF with/out T2D The results demonstrated a 25% reduction in the relative risk of the primary composite EPs of time to CV death or hospitalization for HF. The 2EPs demonstrated 30% …

Boehringer Ingelheim and Eli Lilly’s Jardiance (empagliflozin) Receive the US FDA’s Approval for the Treatment of Heart Failure with Reduced Ejection Fraction Read More »

BeiGene and EUSA Receive the NMPA’s Approval of Qarziba (dinutuximab beta) for High-Risk Neuroblastoma

regulatory / August 18, 2021 August 18, 2021

Shots: The conditional approval is based on the SIOPEN trial evaluating dinutuximab beta vs dinutuximab beta with/out IL-2 in 514 patients aged ≥12 mos. with high-risk neuroblastoma who have received induction CT & partial response along with a history of R/R neuroblastoma with/out the residual disease. The trial was conducted by SIOPEN in collaboration with …

BeiGene and EUSA Receive the NMPA’s Approval of Qarziba (dinutuximab beta) for High-Risk Neuroblastoma Read More »

Agios Reports the US FDA’s Acceptance of NDA and Priority Review for Mitapivat to Treat Pyruvate Kinase Deficiency

Acceptance, Agios, FDA, Mitapivat, NDA, Priority Review, Pyruvate Kinase Deficiency, regulatory, US / August 18, 2021 August 18, 2021

Shots: The NDA & MAA submissions are based on ongoing 2 studies i.e., ACTIVATE & ACTIVATE-T evaluates safety, tolerability & efficacy of mitapivat vs PBO in patients with PKD Results: 40% of patients achieved a hemoglobin response, 33% reduction in transfusion burden @ 24wks. fixed dose period & safety profile was consistent with previously reported …

Agios Reports the US FDA’s Acceptance of NDA and Priority Review for Mitapivat to Treat Pyruvate Kinase Deficiency Read More »

GSK’s Jemperli (dostarlimab-gxly) Receives the US FDA’s Accelerated Approval for the Treatment of dMMR Recurrent or Advanced Solid Tumors

regulatory / August 18, 2021 August 18, 2021

Shots: The approval is based on the results from cohort A1 & F of the ongoing P-I GARNET trial evaluates dostarlimab (500mg, IV, q3w for 4 doses) as monothx. in 209 patients with dMMR solid tumors, including EC & non-endometrial cancer. The therapy was discovered by AnaptysBio & licensed to Tesaro Results: In all dMMR …

GSK’s Jemperli (dostarlimab-gxly) Receives the US FDA’s Accelerated Approval for the Treatment of dMMR Recurrent or Advanced Solid Tumors Read More »

Global regulators seek permanent working group on AI

Artificial Intelligence, digital, digital health, ICMRA, News, regulatory / August 17, 2021 August 17, 2021

Rapid advances in the use of artificial intelligence (AI) in healthcare and medicines development are at risk of outstripping current regulatory frameworks, according to a new report. The International Coalition of Medicines Regulatory Authorities (ICMRA) has recommended that a new permanent working group be set up to keep tabs on the use of AI and …

Global regulators seek permanent working group on AI Read More »

BMS Reports EMA’s Validation of MAA for Opdivo (nivolumab) + Yervoy (ipilimumab) and Opdivo + CT to Treat Esophageal Squamous Cell Carcinoma

BMS, CT, EMA, Esophageal Squamous Cell Carcinoma, ipilimumab, MAA, Nivolumab, Opdivo, regulatory, validation, Yervoy / August 17, 2021 August 17, 2021

Shots: The applications are based on P-III CheckMate -648 trial evaluates Opdivo (3 mg/kg, q2w) + Yervoy (1 mg/kg, q6w up to 24mos.) or Opdivo (nivolumab) + CT vs CT alone in patients with unresectable advanced or metastatic ESCC Results: Both combinations showed OS benefit over CT at the pre-specified interim analysis in patients with …

BMS Reports EMA’s Validation of MAA for Opdivo (nivolumab) + Yervoy (ipilimumab) and Opdivo + CT to Treat Esophageal Squamous Cell Carcinoma Read More »

Novartis’ Cosentyx (secukinumab) Receives the NMPA’s Approval for the Treatment of Pediatric Psoriasis in China

approval, China, Cosentyx, NMPA, Novartis, Pediatric Psoriasis, regulatory, secukinumab / August 17, 2021 August 17, 2021

Shots: The approval is based on two P-III international studies that evaluate cosentyx in pediatric patients aged 6 to <18 yrs. with mod. to sev. PsO who are candidates for systemic therapy or phototherapy The results showed that the therapy is safe and effective for children and adults with PsO. Additionally, therapy has supported the …

Novartis’ Cosentyx (secukinumab) Receives the NMPA’s Approval for the Treatment of Pediatric Psoriasis in China Read More »

Eli Lilly’s Lyumjev (insulin lispro-aabc injection) Receives the US FDA’s Expanded Label Approval for the Treatment of Type 1 and Type 2 Diabetes

Eli Lilly, Expanded Label Approval, FDA, insulin lispro-aabc injection, Lyumjev, regulatory, Type 1 and Type 2 Diabetes, US / August 17, 2021 August 17, 2021

Shots: The approval is based on P-III PRONTO-PUMP-2, a treat-to-target study that evaluates the efficacy & safety of Lyumjev (insulin lispro-aabc injection, 100 units/mL) vs Humalog (insulin lispro injection, 100 units/mL) in 432 adults in a ratio (1:1) with T1D for 16wks. The study met its 1EPs i.e., non-inferior A1C reduction from baseline @16wks. Lyumjev …

Eli Lilly’s Lyumjev (insulin lispro-aabc injection) Receives the US FDA’s Expanded Label Approval for the Treatment of Type 1 and Type 2 Diabetes Read More »

Merck Reports the Initiation of Rolling Submission to Health Canada for Molnupiravir to Treat COVID-19

COVID-19, Health Canada, Merck, molnupiravir, regulatory, Rolling Submission / August 16, 2021 August 16, 2021

Shots: The initiation of a rolling submission to Health Canada for molnupiravir (bid, for 5 days) to treat COVID-19 is based on the P-II interim results from the P-II/III MOVe-OUT clinical trials. The therapy is being developed in collaboration with Ridgeback The submission was accepted under the Minister of Health’s interim order and enables the …

Merck Reports the Initiation of Rolling Submission to Health Canada for Molnupiravir to Treat COVID-19 Read More »

Pfizer’s Ticovac Receives the US FDA’s Approval for the Prevention of Tick-Borne Encephalitis

regulatory / August 16, 2021 August 16, 2021

Shots: The US FDA has approved Ticovac for active immunization to prevent TBE in patients aged ≥1 yrs. The US CDC’s will discuss recommendations regarding the safe and appropriate use of the vaccine In clinical trials, safety & immunogenicity of Ticovac were evaluated across two age groups (aged 1-15 yrs. and >16 yrs.). The studies …

Pfizer’s Ticovac Receives the US FDA’s Approval for the Prevention of Tick-Borne Encephalitis Read More »

The US FDA Rejects Sesen Bio’s Vicineum (oportuzumab monatox-qqrs) for the Treatment of BCG-Unresponsive NMIBC

BCG, FDA, NMIBC, oportuzumab monatox-qqrs, regulatory, Reject, Sesen Bio, Unresponsive, US, Vicineum / August 16, 2021 August 16, 2021

Shots: The company has received the FDA’s CRL declining the approval of Vicineum’s BLA to treat BCG-unresponsive NMIBC The FDA has provided recommendations to conduct additional clinical/statistical data and analyses of roxadustat in addition to CMC issues related to a recent pre-approval inspection and product quality The company plans to meet with the FDA as …

The US FDA Rejects Sesen Bio’s Vicineum (oportuzumab monatox-qqrs) for the Treatment of BCG-Unresponsive NMIBC Read More »

Merck’s Welireg (belzutifan) Receives the US FDA’s Approval for the Treatment of Von Hippel-Lindau Disease Associated Tumors

regulatory / August 16, 2021 August 16, 2021

Shots: The approval is based on open-label study 004 trial evaluates welireg (120 mg, qd) in patients with VHL-associated tumors including RCC, CNS hemangioblastomas, or pNET The results showed ORR (49%), m-DoR had not reached, 56% were still responding after 12mos., m-TTR (8mos.) in 61 patients with VHL-associated RCC, Additionally, 24 & 12 patients with …

Merck’s Welireg (belzutifan) Receives the US FDA’s Approval for the Treatment of Von Hippel-Lindau Disease Associated Tumors Read More »

Junshi and Coherus’s Toripalimab Receive the US FDA’s Breakthrough Therapy Designation as 1L Treatment of Nasopharyngeal Carcinoma

Breakthrough Therapy Designation, Coherus, FDA, Junshi, Nasopharyngeal Carcinoma, regulatory, Toripalimab, US / August 13, 2021 August 13, 2021

Shots: The designation is based on the P-III JUPITER-02 trial evaluating toripalimab + CT vs CT alone in patients with NPC. The result was presented at ASCO 2021 The results demonstrated an improvement in PFS. The trial also meets 2EPs of PFS assessed by the investigator and ORR as assessed by BIRC including higher DoR; …

Junshi and Coherus’s Toripalimab Receive the US FDA’s Breakthrough Therapy Designation as 1L Treatment of Nasopharyngeal Carcinoma Read More »

Celltrion’s Regdanvimab (CT-P59) Receives the ANVISA’s EUA for the Treatment of COVID-19 in Brazil

COVID-19, regulatory / August 13, 2021 August 13, 2021

Shots: The EUA is based on P-III study evaluating regdanvimab vs PBO in adults with COVID-19 who do not require supplemental oxygen & are at high risk of progression to severe COVID-19 The results demonstrated a 72% reduction in risk of COVID-19 related hospitalization or death in high-risk patients & 70% for all patients, shortened …

Celltrion’s Regdanvimab (CT-P59) Receives the ANVISA’s EUA for the Treatment of COVID-19 in Brazil Read More »

Jazz’ Xywav Receives the US FDA’s Approval for the Treatment of Idiopathic Hypersomnia

approval, FDA, Idiopathic Hypersomnia, Jazz, oral solution, regulatory, US, Xywav / August 13, 2021 August 13, 2021

Shots: The approval is based on the P-III withdrawal study that evaluates the efficacy and safety of Xywav (twice or once-nightly regimen, oral solution) vs PBO in adults with IH. The therapy is expected to be available in late 2021, following REMS implementation The results demonstrated the change in the 1EPs of ESS score & …

Jazz’ Xywav Receives the US FDA’s Approval for the Treatment of Idiopathic Hypersomnia Read More »

C4T’s CFT7455 Receives the US FDA’s Orphan Drug designation for the Treatment of Multiple Myeloma

C4T, CFT7455, FDA, multiple myeloma, Orphan Drug Designation, regulatory, US / August 12, 2021 August 12, 2021

Shots: The US FDA has granted ODD to CFT7455 for the treatment of MM. The FDA grants ODD to drugs & biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases, or conditions that affect fewer than 200,000 people in the US In Jun’21, the company has initiated a …

C4T’s CFT7455 Receives the US FDA’s Orphan Drug designation for the Treatment of Multiple Myeloma Read More »

Merck’s Keytruda (pembrolizumab) + Lenvima (lenvatinib) Receive the US FDA’s Approval as 1L Treatment of Advanced Renal Cell Carcinoma

Advanced Renal Cell Carcinoma, approval, FDA, KEYTRUDA, lenvatinib, Lenvima, Merck, pembrolizumab, regulatory, US / August 12, 2021 August 12, 2021

Shots: The approval is based on P-III CLEAR /KEYNOTE-581 trial that evaluates Keytruda (200 mg, IV, q3w) + Lenvima (20 mg, PO, qd) or Lenvima (18mg) + everolimus (5mg, PO, qd) vs sunitinib (50 mg, q4w) in a ratio (1:1:1) in 1,069 patients with advanced RCC The results demonstrated an improvement in PFS with a …

Merck’s Keytruda (pembrolizumab) + Lenvima (lenvatinib) Receive the US FDA’s Approval as 1L Treatment of Advanced Renal Cell Carcinoma Read More »

The US FDA’s Rejects FibroGen’s Roxadustat for the Treatment of Anemia Due to CKD

Anemia, CKD, FDA, FibroGen, regulatory, Reject, Roxadusta, US / August 11, 2021 August 11, 2021

Shots: The company has received an FDA’s CRL declining the approval of roxadustat’s NDA to treat anemia of CKD The FDA has requested to conduct an additional clinical study of roxadustat before resubmission The therapy is approved in China, Japan, Chile, and South Korea for the treatment of anemia of CKD in both NDD and …

The US FDA’s Rejects FibroGen’s Roxadustat for the Treatment of Anemia Due to CKD Read More »

Merck Reports the US FDA’s Acceptance of Keytruda’s sBLA for Review to Treat MSI-H/dMMR Advanced Endometrial Carcinoma

FDA, KEYTRUDA, Merck, MSI-H/dMMR, regulatory, sBLA, US / August 11, 2021 August 11, 2021

Shots: The application is based on ORR data from cohorts D & K of KEYNOTE-158 trial evaluating Keytruda as monothx. (200 mg, q3w) in 90 patients with MSI-H/dMMR advanced EC with disease progression following prior systemic therapy in any setting & are not candidates for curative surgery or radiation This indication is approved under accelerated …

Merck Reports the US FDA’s Acceptance of Keytruda’s sBLA for Review to Treat MSI-H/dMMR Advanced Endometrial Carcinoma Read More »

GW’s Epidyolex (cannabidiol) Receives MHRA’s Approval for the Treatment of Seizures Associated with Tuberous Sclerosis Complex in UK

approval, cannabidiol, Epidyolex, GW, MHRA, regulatory, seizures, Tuberous Sclerosis Complex / August 11, 2021 August 11, 2021

Shots: The approval is based on P-III safety & efficacy study that evaluates Epidyolex (25 mg/kg/day) vs PBO in patients aged ≥2yrs. as an adjunctive treatment with seizures associated with TSC The study met its 1EPs i.e., reduction in seizure frequency (49% vs 27%). The 2EPs had supported the effects on 1EPs & safety profile …

GW’s Epidyolex (cannabidiol) Receives MHRA’s Approval for the Treatment of Seizures Associated with Tuberous Sclerosis Complex in UK Read More »

Merck Reports the US FDA’s Acceptance of sBLA and Priority Review for Keytruda (pembrolizumab) as an Adjuvant Treatment for RCC

FDA, KEYTRUDA, Merck, pembrolizumab, Priority Review, RCC, regulatory, sBLA, US / August 10, 2021 August 10, 2021

Shots: The sBLA is based on P-III KEYNOTE-564 trial evaluating Keytruda monothx. (200mg, IV on day 1 of each 3wks. cycle for ~17 cycles) vs PBO in 994 patients with RCC, following nephrectomy and resection of metastatic lesions The results demonstrated an improvement in DFS and were presented at ASCO 2021 with an anticipated PDUFA …

Merck Reports the US FDA’s Acceptance of sBLA and Priority Review for Keytruda (pembrolizumab) as an Adjuvant Treatment for RCC Read More »

Calliditas Receives the US FDA’s Fast Track Designation for Setanaxib to Treat Primary Biliary Cholangitis

Calliditas, Fast Track Designation, FDA, primary biliary cholangitis, regulatory, Setanaxib, US / August 9, 2021 August 9, 2021

Shots: The US FDA has granted FTD to selinexor (NOX inhibitor) for the treatment of PBC. Additionally, the therapy has previously received ODD for PBC in the US & EU The P-II clinical trial of the therapy demonstrated anti-fibrotic activity as measured by Fibroscan and showed favorable tolerability profile along with the impact on fatigue …

Calliditas Receives the US FDA’s Fast Track Designation for Setanaxib to Treat Primary Biliary Cholangitis Read More »

AstraZeneca’s Forxiga (dapagliflozin) Receives EC’s Approval for the Treatment of Chronic Kidney Disease in Patients with and without T2D

regulatory / August 9, 2021 August 9, 2021

Shots: The approval is based on a P-III DAPA-CKD trial that evaluates the efficacy of Forxiga (10mg, qd in addition to SoC) vs PBO in 4304 patients with CKD Stage 2-4 & elevated urinary albumin excretion with & without T2D The results showed 39% reduction in risk of worsening of renal function, ESKD, CV or …

AstraZeneca’s Forxiga (dapagliflozin) Receives EC’s Approval for the Treatment of Chronic Kidney Disease in Patients with and without T2D Read More »

Sanofi’s Nexviazyme (avalglucosidase alfa-ngpt) Receives the US FDA’s Approval for the Treatment of Late-Onset Pompe Disease

regulatory / August 9, 2021 August 9, 2021

Shots: The approval is based on the P-III COMET trial that evaluates Nexviazyme (q2w, IV infusion) vs alglucosidase alfa in patients aged ≥1yrs. with LOPD for 49wks. Results: patients achieved noninferiority with 2.4 points greater improvement in FVC percent-predicted @49wks., were able to walk 32.2 m farther @49wks. on the 6MWT. The therapy showed improvements …

Sanofi’s Nexviazyme (avalglucosidase alfa-ngpt) Receives the US FDA’s Approval for the Treatment of Late-Onset Pompe Disease Read More »

Pfizer, Flynn in the firing line again over UK epilepsy drug pricing

competition law, drug pricing, Flynn Pharma, News, Pfizer, regulatory, Sales and Marketing, UK / August 5, 2021 August 5, 2021

The UK’s Competition and Markets Authority (CMA) has resurrected a complaint against Pfizer and Flynn Pharma, claiming the two companies charged the NHS excessive prices for a widely-used epilepsy therapy. In a provisional judgment, the CMA has concluded that Pfizer and Flynn abused a dominant position to overcharge the NHS for phenytoin sodium capsules, causing …

Pfizer, Flynn in the firing line again over UK epilepsy drug pricing Read More »

Merck’s Keytruda (pembrolizumab) Receives the US FDA’s Priority Review for the Adjuvant Treatment of Stage II Resected High-Risk Melanoma

KEYNOTE-716 Trial, KEYTRUDA, Merck, P-III, pembrolizumab, regulatory, Resected High-Risk Melanoma, Stage II / August 5, 2021 August 5, 2021

Shots: The priority review is based on P-III KEYNOTE-716 trial assessing Keytruda (anti-PD-1 therapy) vs PBO in 954 patients aged ≥12yrs. with stage II resected high-risk melanoma. The anticipated PDUFA date for the therapy is Dec 04, 2021 The trial met its 1EPs of RFS with no new safety signals observed. Based on this data, …

Merck’s Keytruda (pembrolizumab) Receives the US FDA’s Priority Review for the Adjuvant Treatment of Stage II Resected High-Risk Melanoma Read More »

BeyondSpring Reports Results of Plinabulin + Docetaxel in P-III DUBLIN-3 Trial for 2L and 3L Treatment of NSCLC with EGFR Wild Type

BeyondSpring, Docetaxel, DUBLIN-3 Trial, EGFR Wild Type, NSCLC, P-III, Plinabulin, regulatory / August 4, 2021 August 4, 2021

Shots: The P-III DUBLIN-3 trial evaluates plinabulin + docetaxel vs docetaxel alone in 599 patients with 2L & 3L NSCLC EGFR wild type, with measurable lung lesion The trial met its 1EPs & 2EPs i.e improvement in OS, ORR, PFS, and OS rate @24 & 36mos. (22.1% vs 12.5% & 11.7% vs 5.3%); @48mos. OS …

BeyondSpring Reports Results of Plinabulin + Docetaxel in P-III DUBLIN-3 Trial for 2L and 3L Treatment of NSCLC with EGFR Wild Type Read More »

UCB’s Bimekizumab Receives NICE Recommendation for the Fast-Track Appraisal to Treat Severe Plaque Psoriasis

regulatory / August 3, 2021 August 3, 2021

Shots: NICE has issued a final appraisal determination (FAD) which recommends UCB’s bimekizumab as a treatment option for adults with severe PsO The recommendation is based on four P-III studies i.e BE VIVID, BE RADIANT (P- IIIb), BE READY & BE SURE that demonstrated the superior efficacy and safety of bimekizumab in adults with PsO …

UCB’s Bimekizumab Receives NICE Recommendation for the Fast-Track Appraisal to Treat Severe Plaque Psoriasis Read More »

Roche Reports the US FDA’s Acceptance of BLA and Priority Review of Tecentriq (atezolizumab) as an Adjuvant Treatment for NSCLC

atezolizumab, BLA, FDA, NSCLC, Priority Review, regulatory, Roche, Tecentriq, US / August 3, 2021 August 3, 2021

Shots: The application is based on the P-III IMpower010 study evaluating the efficacy & safety of Tecentriq vs BSC in 1005 patients in a ratio (1:1) with Stage IB-IIIA NSCLC, following surgical resection and up to 4 cycles of adjuvant cisplatin-based CT The results from an interim analysis showed the reduction in the risk of …

Roche Reports the US FDA’s Acceptance of BLA and Priority Review of Tecentriq (atezolizumab) as an Adjuvant Treatment for NSCLC Read More »

Janssen’s Uptravi (selexipag) Receives the US FDA’s Approval for IV Use in Adults with Pulmonary Arterial Hypertension

approval, FDA, Janssen, Pulmonary Arterial Hypertension, regulatory, selexipag, UPTRAVI, US / August 2, 2021 August 2, 2021

Shots: The approval is based on P-III UPTRAVI IV study evaluating the safety, tolerability, and PK of temporarily switching Uptravi (PO) to Uptravi (IV) in 20 patients with PAH The results showed that switching b/w Uptravi (PO) to Uptravi IV was well tolerated with no unexpected safety findings. Both formulations maintain the treatment effect for …

Janssen’s Uptravi (selexipag) Receives the US FDA’s Approval for IV Use in Adults with Pulmonary Arterial Hypertension Read More »

AstraZeneca’s Saphnelo (anifrolumab-fnia) Receives the US FDA’s Approval for the Treatment of Moderate to Severe Systemic Lupus Erythematosus

anifrolumab-fnia, approval, AstraZeneca, FDA, regulatory, Saphnelo, Systemic Lupus Erythematosus, US / August 2, 2021 August 2, 2021

Shots: The approval is based on efficacy and safety data from the clinical development program, including two P- III TULIP trials and the P-II MUSE trial evaluating Saphnelo vs PBO in patients with SLE who are receiving standard therapy The result from the trials showed a reduction in overall disease activity across organ systems, including …

AstraZeneca’s Saphnelo (anifrolumab-fnia) Receives the US FDA’s Approval for the Treatment of Moderate to Severe Systemic Lupus Erythematosus Read More »

GSK’s Nucala (mepolizumab) Receives the US FDA’s Approval for the Treatment of Chronic Rhinosinusitis with Nasal Polyps

Chronic Rhinosinusitis, FDA, GSK, mepolizumab, Nucala, regulatory, US / July 30, 2021 July 30, 2021

Shots: The approval is based on data from the SYNAPSE study evaluating mepolizumab vs PBO in ~400 patients with CRSwNP who had a history of prior surgery & need further surgery due to severe symptoms & increased size of polyps The results showed a 57% reduction in the size of nasal polyps & nasal obstruction …

GSK’s Nucala (mepolizumab) Receives the US FDA’s Approval for the Treatment of Chronic Rhinosinusitis with Nasal Polyps Read More »

UK fines Advanz £100m for raising thyroid drug price tenfold

Advanz Pharma, competition, competition law, News, regulatory, Sales and Marketing, UK / July 29, 2021 July 29, 2021

The UK has levied another big fine for anticompetitive activity in the pharma market in a fortnight, with Advanz Pharma and former owners on the hook for more than £100 million ($140 million) after increasing the price of a thyroid disease drug by 1,110% over an eight-year period. The Competition and Markets Authority (CMA) said …

UK fines Advanz £100m for raising thyroid drug price tenfold Read More »

Takeda’s TAK-994 Receives Breakthrough Therapy Designation for the Treatment of Narcolepsy Type 1

Breakthrough Therapy Designation, Narcolepsy Type, regulatory, TAK-994, Takeda / July 29, 2021 July 29, 2021

Shots: The BTD is based on early and preliminary clinical data involves assessing TAK-994 for the treatment of EDS that may demonstrated substantially improved objective and subjective measurements of daytime wakefulness in NT1 patients TAK-994 is being evaluated in an ongoing P-II TAK-994-1501 study while the completed data from study will be presented at a …

Takeda’s TAK-994 Receives Breakthrough Therapy Designation for the Treatment of Narcolepsy Type 1 Read More »

Roche Reports the US FDA’s Acceptance of BLA for Faricimab to Treat Diabetic Macular Edema and Neovascular Age-Related Macular Degeneration

Acceptance, BLA, Diabetic Macular Edema, Faricimab, FDA, Neovascular Age-Related Macular Degeneration, regulatory, Roche, US / July 29, 2021 July 29, 2021

Shots: The BLA submission is based on four P-III studies i.e TENAYA, LUCERNE, YOSEMITE & RHINE evaluating the efficacy & safety of faricimab vs aflibercept in patients with nAMD & DME. The LTE studies for faricimab are underway The studies demonstrated non-inferior visual acuity gains and greater reductions in CST, ~50% of patients can extend …

Roche Reports the US FDA’s Acceptance of BLA for Faricimab to Treat Diabetic Macular Edema and Neovascular Age-Related Macular Degeneration Read More »

Sanofi’s Avalglucosidase alfa Receives CHMP’s Positive Opinion for Patients with Pompe Disease

Avalglucosidase-alfa, CHMP, Pompe disease, Positive Opinion, regulatory, Sanofi / July 28, 2021 July 28, 2021

Shots: The CHMP’s positive opinion is based on P-III COMET study assessing avalglucosidase alfa that demonstrated improvements in respiratory function & movement endurance measures in people with late-onset Pompe disease The CHMP concluded that avalglucosidase alfa does not qualify as a New Active Substance (NAS). Sanofi will be requesting a re-examination of the CHMP’s opinion …

Sanofi’s Avalglucosidase alfa Receives CHMP’s Positive Opinion for Patients with Pompe Disease Read More »

GSK’s Shingrix Receives the US FDA’s Approval to Prevent Shingles in Immunocompromised Adults

approval, FDA, GSK, Immunocompromised Adults, regulatory, Shingles, Shingrix, US / July 27, 2021 July 27, 2021

Shots: The approval is based on studies evaluating the safety & efficacy of Shingrix in adults aged ≥18yrs. who had undergone auHSCT and those undergoing treatment for hematological malignancies (post-hoc analysis) The safety & immunogenicity data were generated in adults who were or anticipated to be, immunodeficient/immunosuppressed due to known therapy including patients with HIV, …

GSK’s Shingrix Receives the US FDA’s Approval to Prevent Shingles in Immunocompromised Adults Read More »

BeiGene’s Brukinsa (Zanubrutinib) Receives Health Canada’s Approval for Mantle Cell Lymphoma

regulatory / July 27, 2021 July 27, 2021

Shots: The approval is based on efficacy results from two studies. The P-II BGB-3111-206 study evaluates zanubrutinib in patients with R/R MCL and showed ORR (84%), CR (69%), PR (15%), mDoR (19.5mos.) @median follow-up time of 18.5mos. The P-I/II BGB-3111-AU-003 study showed ORR (84%) including CR (25%) & PRs (59%), m-DoR (18.5mos.) @ median follow-up …

BeiGene’s Brukinsa (Zanubrutinib) Receives Health Canada’s Approval for Mantle Cell Lymphoma Read More »

The US FDA Declines to Approve Incyte’s Retifanlimab to Treat Patients with Squamous Cell Carcinoma of the Anal Canal

Anal Canal, Approve, Declines, FDA, Incyte, regulatory, Retifanlimab, Squamous Cell Carcinoma, US / July 26, 2021 July 26, 2021

Shots: The US FDA has issued a CRL to the BLA of retifanlimab. The BLA submission is based on P-II POD1UM-202 trial assessing retifanlimab in prior treated patients with LA or m-SCAC who have progressed on or were ineligible for or intolerant of Pt. based CT The FDA stated that an additional data is needed …

The US FDA Declines to Approve Incyte’s Retifanlimab to Treat Patients with Squamous Cell Carcinoma of the Anal Canal Read More »

Alexion’s Ultomiris (ravulizumab) Receives CHMP’s Positive Opinion Recommending its Approval for Children and Adolescents with PNH

Adolescents, Alexion, approval, Children, CHMP, PNH, Positive Opinion, ravulizumab, receives, Recommending, regulatory, Ultomiris / July 26, 2021 July 26, 2021

Shots: The CHMP’s positive opinion is based on a P-III study assessing Ultomiris and showed effectiveness in completing C5 complement inhibition @26wks. for children & adolescents aged ≤18yrs. with PNH The study demonstrated that there is no reported treatment-related sAEs, and no patients discontinued treatment during the primary evaluation period or experienced breakthrough hemolysis The …

Alexion’s Ultomiris (ravulizumab) Receives CHMP’s Positive Opinion Recommending its Approval for Children and Adolescents with PNH Read More »

AstraZeneca’s Bydureon BCise (exenatide extended-release) Receives the US FDA’s Approval for the Treatment of Type2 Diabetes

regulatory / July 23, 2021 July 23, 2021

Shots: The approval is based on results of the P-III BCB114 trial evaluating Bydureon BCise (qw, injectable suspension) vs PBO in 82 patients aged b/w 10 – 18 yrs. with T2D as an adjuvant to diet and exercise alone or in combination with a stable dose of oral antidiabetic agents and insulin for 24 wks. …

AstraZeneca’s Bydureon BCise (exenatide extended-release) Receives the US FDA’s Approval for the Treatment of Type2 Diabetes Read More »

Merck and Eisai’s Keytruda (pembrolizumab) + Lenvima (lenvatinib) Receive the US FDA’s Approval for the Treatment of Advanced Endometrial Carcinoma

regulatory / July 22, 2021 July 22, 2021

Shots: The approval is based on P-III KEYNOTE-775/Study 309 trial evaluating Merck’s Keytruda (200mg, IV, q3w) + Eisai’s Lenvima (20mg, PO, qd) vs CT (doxorubicin, 60 mg/m2, IV, q3wks.) or paclitaxel (80 mg/m2, IV, 28-day cycle) in 827 patients with advanced EC that were not MSI-H or dMMR, prior treated with Pt-based regimen in any …

Merck and Eisai’s Keytruda (pembrolizumab) + Lenvima (lenvatinib) Receive the US FDA’s Approval for the Treatment of Advanced Endometrial Carcinoma Read More »

Bluebird Bio’s Skysona (elivaldogene autotemcel) Receives EC’s Approval for the Treatment of Cerebral Adrenoleukodystrophy

regulatory / July 22, 2021 July 22, 2021

Shots: The approval is based on P-II/III ALD-102 study evaluating Skysona in patients with an ABCD1 gene mutation aged <18 yrs. with CALD without matched sibling donors. The P-III ALD-104 study is ongoing Results: 90% of patients met the MFD-free survival 1EPs @24mos., 26 out of 28 patients maintained an NFS ≤1 @24mos. All patients …

Bluebird Bio’s Skysona (elivaldogene autotemcel) Receives EC’s Approval for the Treatment of Cerebral Adrenoleukodystrophy Read More »

Mabpharm’s Biobetter (CMAB008) for Infliximab Receives NMPA’s Approval in China

Biosimilars, regulatory / July 22, 2021 July 22, 2021

Shots: NMPA has granted approval to market infliximab biobetter in China. Sorrento holds exclusive commercial rights to Infliximab biobetter Ab outside of China and plans to file a BLA for the product in the US and EU in 2021 Biobetters are drugs that have similar structure and properties to an innovator product but are enhanced …

Mabpharm’s Biobetter (CMAB008) for Infliximab Receives NMPA’s Approval in China Read More »

Bayer and Merck’s Verquvo (vericiguat) Receives the EC’s Approval for the Treatment of Chronic Heart Failure

regulatory / July 22, 2021 July 22, 2021

Shots: The EC has approved Verquvo (2.5/5/10 mg) in the EU for the treatment of symptomatic CHF in adult patients with reduced ejection fraction who are stabilized after a recent decompensation event. The therapy is being jointly developed by both companies Merck has the commercial rights to Verquvo in the US and Bayer has the …

Bayer and Merck’s Verquvo (vericiguat) Receives the EC’s Approval for the Treatment of Chronic Heart Failure Read More »

Genentech’s Venclexta (venetoclax) + Azacitidine Receives Breakthrough Therapy Designation for the Treatment of Myelodysplastic Syndromes

regulatory / July 21, 2021 July 21, 2021

Shots: The designation is based on interim results from the P-Ib M15-531 study evaluating Venclexta + azacitidine in naïve patients with previously untreated higher-risk MDS The primary objectives of the study are to assess the safety, Pk and determine the RP2D and dosing schedule of Venclexta in combination with azacitidin Venclexta is a novel inhibitor …

Genentech’s Venclexta (venetoclax) + Azacitidine Receives Breakthrough Therapy Designation for the Treatment of Myelodysplastic Syndromes Read More »

Albireo’s Bylvay (odevixibat) Receives the US FDA’s Approval for the Treatment of Progressive Familial Intrahepatic Cholestasis

regulatory / July 21, 2021 July 21, 2021

Shots: The approval is based on P-III PEDFIC 1 & 2 studies evaluating Bylvay (qd) vs PBO in patients with pruritus in PFIC. The PEDFIC 1 study met its 1EPs i.e the therapy showed a low incidence of diarrhea or frequent bowel movements (9.5% vs 5.0%) The PEDFIC 2 study showed sustained reductions in serum …

Albireo’s Bylvay (odevixibat) Receives the US FDA’s Approval for the Treatment of Progressive Familial Intrahepatic Cholestasis Read More »

Myovant’s Ryeqo (relugolix, estradiol, and norethindrone acetate) Receives EC’s Approval for the Treatment of Women with Uterine Fibroids

approval, EC, estradiol, Myovant, norethindrone acetate, regulatory, Relugolix, Ryeqo, Uterine Fibroids / July 20, 2021 July 20, 2021

Shots: The approval is based on safety & efficacy data from the P-III LIBERTY program that consists of 2 studies i.e LIBERTY 1 & 2 evaluating Ryeqo in adult women with UF with no limitation for a duration of use. The EC’s decision is valid in all 27 member states of the EU, Iceland, Norway …

Myovant’s Ryeqo (relugolix, estradiol, and norethindrone acetate) Receives EC’s Approval for the Treatment of Women with Uterine Fibroids Read More »

FDA gives speedy review to Bayer’s Parkinson’s stem cell therapy

Bayer, BlueRock, Market Access, neurology, News, Parkinson’s Disease, regulatory, stem cell / July 20, 2021 July 20, 2021

Bayer subsidiary BlueRock Therapeutics has been granted a fast-track review by the FDA for DA01, its stem cell-based therapy for Parkinson’s disease which is currently in early-stage clinical testing. The FDA designation allows for benefits such as more frequent meetings and communication with the regulator during clinical development, and a truncated six-month review time. Those …

FDA gives speedy review to Bayer’s Parkinson’s stem cell therapy Read More »

Kadmon’s Rezurock (belumosudil) Receives the US FDA’s Approval for the Treatment of Chronic Graft Versus Host Disease

approval, belumosudil, FDA, Kadmon, regulatory, Rezurock, US / July 20, 2021 July 20, 2021

Shots: The approval is based on KD025-213 study evaluating Rezurock (200 mg, qd) aged >12 yrs. in 65 patients with cGVHD who had received 2 to 5 prior lines of systemic therapy. The anticipated PFUFA date is Aug 30, 2021 Results: ORR (75%) through Cycle 7, day 1 treatment with CR (6%) & PR (69%); …

Kadmon’s Rezurock (belumosudil) Receives the US FDA’s Approval for the Treatment of Chronic Graft Versus Host Disease Read More »

Regeneron’s Ronapreve Antibody Cocktail (casirivimab and imdevimab) Receives MHLW’s Approval for the Treatment of Mild to Moderate COVID-19

approval, Casirivimab, COVID-19, Imdevimab, MHLW, REGEN-COV Antibody Cocktail, Regeneron, regulatory / July 20, 2021 July 20, 2021

Shots: The approval is based on the results from a P-III trial evaluating Ronapreve (casirivimab and imdevimab) in high-risk non-hospitalized patients with COVID-19 The results showed a reduction in the risk of hospitalization or death by 70% in addition to the results from a P-I trial that demonstrated the safety, tolerability & PK in Japanese …

Regeneron’s Ronapreve Antibody Cocktail (casirivimab and imdevimab) Receives MHLW’s Approval for the Treatment of Mild to Moderate COVID-19 Read More »

AstraZeneca’s Imfinzi (durvalumab) Receives NMPA’s Approval as a 1L Treatment of Extensive-Stage Small Cell Lung Cancer

regulatory / July 19, 2021 July 19, 2021

Shots: The approval is based on P-III CASPIAN trial assessing Imfinzi + SoC (etoposide & carboplatin/ cisplatin CT) or Imfinzi + CT + tremelimumab vs CT alone in 805 patients with ES-SCLC in 200 centers across 23 countries Results: the study met its 1EPs of OS in Jun’19, demonstrating a 27% reduction in risk of …

AstraZeneca’s Imfinzi (durvalumab) Receives NMPA’s Approval as a 1L Treatment of Extensive-Stage Small Cell Lung Cancer Read More »

Sanofi’s Fexinidazole Receives the US FDA’s Approval as the First Oral Treatment for Sleeping Sickness

regulatory / July 19, 2021 July 19, 2021

Shots: The US FDA has approved fexinidazole (PO, qd, 10days treatment) for both stages of Trypanosoma brucei gambiense form of sleeping sickness in patients aged >6yrs. & weighing at least 20 kg. The therapy has developed under the collaboration with DNDi, DRC, CAR & Sanofi Sanofi & partners are committed to access fexinidazole in all …

Sanofi’s Fexinidazole Receives the US FDA’s Approval as the First Oral Treatment for Sleeping Sickness Read More »

Merck’s Vaxneuvance (pneumococcal 15-valent conjugate vaccine) Receives the US FDA’s Approval for the Prevention of Invasive Pneumococcal Disease Caused by 15 Serotypes

15 Serotypes, approval, Biotech, FDA, Invasive Pneumococcal Disease, Merck, Pneumococcal 15-valent Conjugate Vaccine, regulatory, US, Vaxneuvance / July 19, 2021 July 19, 2021

Shots: The approval is based on seven P-II & III clinical studies involves assessing the safety, tolerability & immunogenicity of Vaxneuvance vs PCV13 in 7,438 adults aged >18yrs. with IPD caused by 15 serotypes Results: Vaxneuvance showed a non-inferior immune response to PCV13 for 13 shared serotypes as assessed by OPA GMTs & superior immune …

Merck’s Vaxneuvance (pneumococcal 15-valent conjugate vaccine) Receives the US FDA’s Approval for the Prevention of Invasive Pneumococcal Disease Caused by 15 Serotypes Read More »

UK fines drugmakers record £260m for price gouging NHS

competition, News, pricing, regulatory, Sales and Marketing, UK / July 15, 2021 July 15, 2021

The UK’s competition authority has issued its largest ever fine of more than £260 million ($360 million) to several pharma companies accused of colluding to hike the price of medicines delivered to the NHS. The Competition and Markets Authority (CMA) took the action against Auden Mckenzie and Actavis UK – now known as Accord-UK – …

UK fines drugmakers record £260m for price gouging NHS Read More »

FDA staffers defend Aduhelm approval in JAMA editorial

aducanumab, Aduhelm, alzheimer's disease, Biogen, central nervous system, Eisai, FDA, neurology, News, regulatory / July 14, 2021 July 14, 2021

Three FDA figures at the centre of the fallout over the approval Biogen and Eisai’s Alzheimer’s disease therapy Aduhelm have defended their actions, arguing that to do otherwise would have left millions of patients in limbo for years. The trio say that the decision to approve Aduhelm (aducanumab) was extremely challenging as “evidence was strongly …

FDA staffers defend Aduhelm approval in JAMA editorial Read More »

Final hurdle falls as UK backs AZ’s $39bn takeover of Alexion

Alexion, AstraZeneca, Financial, immunology, M&A, News, rare disease, regulatory, Sales and Marketing / July 14, 2021 July 14, 2021

The UK’s competition regulator has approved AstraZeneca’s $39 billion acquisition of US-based drugmaker Alexion, without opting for a broader inquiry into the deal. The nod from the Competition and Markets Authority (CMA) after an initial assessment means that it concluded no antitrust risks were associated with the deal. It was the final needed from a …

Final hurdle falls as UK backs AZ’s $39bn takeover of Alexion Read More »

Janssen’s Darzalex Faspro (daratumumab and hyaluronidase-fihj) + Pomalidomide and Dexamethasone Receive the US FDA’s Approval to Treat Patients with Multiple Myeloma

approval, daratumumab, Darzalex Faspro, Dexamethasone, FDA, hyaluronidase-fihj, Janssen, multiple myeloma, pomalidomide, regulatory, US / July 13, 2021 July 13, 2021

Shots: The approval is based on P-III APOLLO study evaluating Darzalex Faspro (SC) + pomalidomide & dexamethasone (Pd) vs Pd alone in 304 patients with RRMM who have received at least one prior treatment regimen including lenalidomide & a proteasome inhibitor The study met its 1EPs of PFS & showed 37% reduction in risk of …

Janssen’s Darzalex Faspro (daratumumab and hyaluronidase-fihj) + Pomalidomide and Dexamethasone Receive the US FDA’s Approval to Treat Patients with Multiple Myeloma Read More »

Astellas and Seagen’s Padcev (enfortumab vedotin-ejfv) Receive the US FDA’s Full Approval and Expands Indication for Patients with Locally Advanced or Metastatic Urothelial Cancer

regulatory / July 12, 2021 July 12, 2021

Shots: The approval is based on P-III EV-301 trial evaluating Padcev vs CT in 608 patients with LA or mUC who were previously treated with Pt.-based CT and a PD-1/L1 inhibitor In a pre-specified interim analysis, patients who received Padcev have m-OS (12.9 vs 9.0 mos.) i.e. 3.9mos. longer than those who received CT Padcev …

Astellas and Seagen’s Padcev (enfortumab vedotin-ejfv) Receive the US FDA’s Full Approval and Expands Indication for Patients with Locally Advanced or Metastatic Urothelial Cancer Read More »

Bayer’s Kerendia (finerenone) Receives the US FDA’s Approval for the Treatment of Chronic Kidney Disease Associated with Type 2 Diabetes

Bayer, finerenone, Kerendia, regulatory / July 12, 2021 July 12, 2021

Shots: The approval is based on a P-III FIDELIO-DKD study evaluating finerenone (10/20mg) + SoC vs PBO + SoC in ~ 5700 patients with CKD & T2D. Patients were followed for a median of 2.6yrs. Results: reduction in the incidence of the primary composite EPs of a sustained decline in eGFR of ≥40%, reduction in …

Bayer’s Kerendia (finerenone) Receives the US FDA’s Approval for the Treatment of Chronic Kidney Disease Associated with Type 2 Diabetes Read More »

Lysogene Receives the US FDA’s Fast Track Designation for LYS-GM101 Gene Therapy to Treat GM1 Gangliosidosis

regulatory / July 9, 2021 July 9, 2021

Shots: The FDA has granted FTD to LYS-GM101 for the Treatment of GM1 Gangliosidosis. The company has recently initiated an adaptative-design clinical trial evaluating the efficacy & safety of LYS-GM101 in 16 patients with GM1 gangliosidosis across the US & EU The P-II/III clinical trial for MPS IIIA is ongoing, following collaboration with Sarepta whereas …

Lysogene Receives the US FDA’s Fast Track Designation for LYS-GM101 Gene Therapy to Treat GM1 Gangliosidosis Read More »

Jazz’s Vyxeos (daunorubicin and cytarabine liposome) for Injection Receives Health Canada Approval for the Treatment of High-risk Acute Myeloid Leukemia

regulatory / July 9, 2021 July 9, 2021

Shots: The approval is based on the results from a P-III study that involves assessing Vyxeos (IV infusion) vs a current conventional treatment group in 309 adult patients aged 60-75 yrs. with newly diagnosed t-AML or AML-MRC The study met its 1EPs i.e the therapy demonstrates the improvement in OS; m-OS (9.6 vs 6.0 mos.) …

Jazz’s Vyxeos (daunorubicin and cytarabine liposome) for Injection Receives Health Canada Approval for the Treatment of High-risk Acute Myeloid Leukemia Read More »

BeiGene’s Kyprolis (carfilzomib) Receives NMPA’s Conditional Approval for the Treatment of Relapsed or Refractory Multiple Myeloma

BeiGene, carfilzomib, Conditional Approval, KYPROLIS, multiple myeloma, NMPA, regulatory / July 9, 2021 July 9, 2021

Shots: The approval is based on P-III trial evaluating efficacy & safety of Kyprolis + dexamethasone in 123 patients with RRMM who have received at least 2 prior therapies, including a proteasome inhibitor & an immunomodulatory agent Results: ORR (35.8%); m-PFS (5.6mos.) as assessed by IRC. Additionally, results showed that the efficacy & safety in …

BeiGene’s Kyprolis (carfilzomib) Receives NMPA’s Conditional Approval for the Treatment of Relapsed or Refractory Multiple Myeloma Read More »

AstraZeneca and Amgen Report the US FDA’s Acceptance of BLA and Priority Review of Tezepelumab for the Treatment of Asthma

Amgen, Asthma, AstraZeneca, BLA, FDA, Priority Review, regulatory, Tezepelumab, US / July 8, 2021 July 8, 2021

Shots: The BLA is based on PATHFINDER clinical program, including P-III NAVIGATOR study evaluating Tezepelumab + SOC vs PBO + SOC in 1,061 adults (18–80yrs.) & adolescents (12–17yrs.) with severe & uncontrolled asthma who receive treatment with medium/high dose ICS + at least 1 additional controller medication with/out OCS The results demonstrated superiority in 1EP …

AstraZeneca and Amgen Report the US FDA’s Acceptance of BLA and Priority Review of Tezepelumab for the Treatment of Asthma Read More »

BeiGene Reports NMPA’s Acceptance of sBLA for Tislelizumab to Treat Esophageal Squamous Cell Carcinoma in China

BeiGene, China, Esophageal Squamous Cell Carcinoma, NMPA, regulatory, sBLA, Tislelizumab / July 8, 2021 July 8, 2021

Shots: The sBLA is based on P-III RATIONALE 302 trial evaluating efficacy & safety of tislelizumab vs CT as a 2L treatment in 512 patients in a ratio (1:1) with locally advanced or metastatic ESCC across 11 countries The 1EPs of a trial is OS in ITT populations & 2EPs is OS in patients with …

BeiGene Reports NMPA’s Acceptance of sBLA for Tislelizumab to Treat Esophageal Squamous Cell Carcinoma in China Read More »

Y-mAbs Reports BLA Submission to the NMPA for Danyelza (naxitamab-gqgk) to Treat Relapsed/Refractory High-Risk Neuroblastoma

(naxitamab-gqgk), BLA, China, Danyelza, High-Risk Neuroblastoma, NMPA, regulatory, Y-mAbs / July 7, 2021 July 7, 2021

Shots: SciClone has submitted the BLA for Danyelza to treat pediatric patients aged >1 yr. and adult patients with r/r high-risk neuroblastoma to NMPA in China If approved, the therapy will be available in the China imminently. The therapy was approved under accelerated approval based on ORR and DoR while the continued approval for the …

Y-mAbs Reports BLA Submission to the NMPA for Danyelza (naxitamab-gqgk) to Treat Relapsed/Refractory High-Risk Neuroblastoma Read More »

Merck’s Keytruda (pembrolizumab) Receives the US FDA’s Approval for Expanded Indication in Patients with Locally Advanced Cutaneous Squamous Cell Carcinoma

approval, Cutaneous Squamous Cell Carcinoma, FDA, KEYTRUDA, Locally Advanced, Merck, pembrolizumab, regulatory, US / July 6, 2021 July 6, 2021

Shots: The approval is based on data from the second interim analysis of the P-II KEYNOTE-629 trial assessing Keytruda (200mg, IV, q3w) in 159 patients with recurrent, metastatic, or LA cSCC that is not curable by surgery or radiation Results: ORR (50%); CR (17%); PR (33%), 81% had DOR of 6mos. or longer, and 37% …

Merck’s Keytruda (pembrolizumab) Receives the US FDA’s Approval for Expanded Indication in Patients with Locally Advanced Cutaneous Squamous Cell Carcinoma Read More »

Immunic’s IMU-838 Receives the US FDA’s IND Approval to Initiate P-III ENSURE & P-II CALLIPER Studies for RRMS & PMS

Clinical Trials, regulatory / July 6, 2021 July 6, 2021

Shots: The P-III ENSURE (1 &2) program evaluates IMU-838 (30mg, qd) vs PBO in ~ 1,050 patients with active RMS. The 1EPs of both trials is time to 1st relapse @72wks. & 2EPs include a volume of T2-lesions, time to confirm disability progression, changes in cognition & measure brain volume The P-II CALLIPER trial assess …

Immunic’s IMU-838 Receives the US FDA’s IND Approval to Initiate P-III ENSURE & P-II CALLIPER Studies for RRMS & PMS Read More »

Lupin’s Solosec (secnidazole) Receives the US FDA’s Approval for the Treatment of Trichomoniasis

regulatory / July 5, 2021 July 5, 2021

Shots: The approval is based on a P-III clinical trial that involves assessing Solosec (2g, PO, granules) vs PBO in 64 patients with trichomoniasis The P-III trial demonstrated 92.2% cure rate in the modified ITT population and 94.9% in the per-protocol population, therapy was well-tolerated with no serious AEs were identified in the trial Additionally, …

Lupin’s Solosec (secnidazole) Receives the US FDA’s Approval for the Treatment of Trichomoniasis Read More »

Hutchmed Reports the US FDA’s Acceptance of Surufatinib’s NDA for Advanced Neuroendocrine Tumors

Acceptance, Advanced Neuroendocrine Tumors, FDA, Hutchmed, NDA, regulatory, Surufatinib, US / July 2, 2021 July 2, 2021

Shots: The NDA is based on two P-III studies of surufatinib in patients with pancreatic and extra-pancreatic NET in China. The anticipated PDUFA date is April 30, 2022 The same data from the studies will be used to file MAA to the EMA imminently, based on scientific advice from the EMA’s Committee for Medicinal Products …

Hutchmed Reports the US FDA’s Acceptance of Surufatinib’s NDA for Advanced Neuroendocrine Tumors Read More »

Zydus Seeks EUA for ZyCoV-D Against COVID-19 in India

COVID-19, EUA, India, regulatory, ZyCoV-D, Zydus / July 2, 2021 July 2, 2021

Shots: The company has applied for emergency use authorization to the DCGI for ZyCoV-D, which is the world’s first plasmid DNA vaccine for COVID-19 Zydus conducted a clinical trial for its ZyCoV-D in the adolescent population aged 12-18yrs. in India over 50 centers. The vaccine is safe and well-tolerated and showed 66% efficacy for symptomatic …

Zydus Seeks EUA for ZyCoV-D Against COVID-19 in India Read More »

The US FDA Approves FoundationOne CDx for Alunbrig (brigatinib) in Patients with ALK+ NSCLC

ALK+, Alunbrig, brigatinib, CDx, FDA, FoundationOne CDx, MedTech, NSCLC, regulatory, US / July 2, 2021 July 2, 2021

Shots: The US FDA has approved FoundationOne CDx to be used as a CDx for Alunbrig (brigatinib) which is approved for the treatment of adult patients with ALK+ mNSCLC as detected by an FDA-approved test In Sept’20, Foundation Medicine and Takeda collaborated to develop CDx for therapies in its late-stage lung portfolio FoundationOne CDx is …

The US FDA Approves FoundationOne CDx for Alunbrig (brigatinib) in Patients with ALK+ NSCLC Read More »

Jazz’s Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn) Receives the US FDA’s Approval for Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma

Acute Lymphoblastic Leukemia, approval, asparaginase erwinia chrysanthemi, FDA, Jazz, Lymphoblastic Lymphoma, regulatory, Rylaze, US / July 1, 2021 July 1, 2021

Shots: The approval is based on clinical data from an ongoing P-II/III JZP458-201 trial evaluating Rylaze in pediatric and adult patients aged ≥1mos. with ALL or LBL who are allergic to the E. coli-derived asparaginase. Rylaze has approved under the RTOR program Additionally, recent data from a Children’s Oncology Group retrospective analysis in ~8,000 patients …

Jazz’s Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn) Receives the US FDA’s Approval for Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma Read More »

Abbott’s XIENCE Stent Receives the US FDA’s Approval for Shortest Blood Thinner Course in Patients with High Bleeding Risk

MedTech, regulatory / July 1, 2021 July 1, 2021

Shots: Abbott has received approval in the US & and CE mark for XIENCE stents, the shortest approved DAPT labeling as short as 28 days in patients with high bleeding risk. XIENCE Skypoint stent also received FDA approval in the US and CE Mark in the EU Additionally, patients with stents are typically on DAPT …

Abbott’s XIENCE Stent Receives the US FDA’s Approval for Shortest Blood Thinner Course in Patients with High Bleeding Risk Read More »

LEXEO Therapeutics’ LX2006 Receives the US FDA’s Rare Pediatric Disease Designation and Orphan Drug Designation to Treat Friedreich Ataxia

Friedreich Ataxia, LEXEO Therapeutics, LX2006, Orphan Drug Designation, Rare Pediatric Disease Designation, regulatory / July 1, 2021 July 1, 2021

Shots: The US FDA has granted RPD and ODD to LX2006 for the treatment of FA. The company plans to start a P-I/II clinical trial of LX2006 in patients with cardiomyopathy associated with FA in 2021 Additionally, if BLA for LX2006 is approved, a company may be eligible to receive PRV that may be sold …

LEXEO Therapeutics’ LX2006 Receives the US FDA’s Rare Pediatric Disease Designation and Orphan Drug Designation to Treat Friedreich Ataxia Read More »

Formycon and Bioeq Report MAA Submission to EMA for FYB201 (biosimilar, ranibizumab)

Biosimilars, regulatory / June 30, 2021 June 30, 2021

Shots: Formycon and Bioeq have submitted MAA for FYB201 (biosimilar referencing Lucentis) to EMA Teva will responsible for the commercialization of FYB201 in the EU which has acquired the distribution rights under its collaboration with Bioeq. Currently, Formycon has four biosimilars in development Formycon focuses to treat ophthalmology, immunology, and other key chronic diseases covering …

Formycon and Bioeq Report MAA Submission to EMA for FYB201 (biosimilar, ranibizumab) Read More »

Blueprint Medicines’ Ayvakit (avapritinib) Receives FDA’s Approval to Treat Patients with Advanced Systemic Mastocytosis

Advanced Systemic Mastocytosis, approval, avapritinib, Ayvakit, blueprint-medicines, FDA, regulatory / June 17, 2021 June 17, 2021

Shots: The full FDA approval is based on data from 2 clinical trials i.e., P-I EXPLORER & P-II PATHFINDER trial evaluating the efficacy of Ayvakit (200 mg, PO, qd) in 53 adults with advanced SM including ASM, SM-AHN & MCL Results: @median follow-up of 11.6 mos., ORR (57%), CR/CRh (28%), PR (28 %) & clinical …

Blueprint Medicines’ Ayvakit (avapritinib) Receives FDA’s Approval to Treat Patients with Advanced Systemic Mastocytosis Read More »

MannKind and United Therapeutics Reports the US FDA’s Acceptance of NDA for Priority Review of Tyvaso DPI to Treat PAH and PH-ILD

Acceptance, FDA, MannKind, NDA, PAH, PH-ILD, Priority Review, regulatory, Tyvaso DPI, United Therapeutics, US / June 17, 2021 June 17, 2021

Shots: The NDA is based on data from the BREEZE study evaluating Tyvaso DPI in 51 patients with PAH transitioning from Tyvaso inhalation solution which demonstrates the safety & tolerability of Tyvaso DPI Additionally, a separate PK study in 36 healthy volunteers demonstrates comparable treprostinil exposure b/w Tyvaso DPI & Tyvaso Inhalation Solution while FDA …

MannKind and United Therapeutics Reports the US FDA’s Acceptance of NDA for Priority Review of Tyvaso DPI to Treat PAH and PH-ILD Read More »

BeiGene’s Tislelizumab in Combination with Asieris’ APL-1202 Receive the US FDA’s IND Approval for MIBC

APL-1202, Asieris, BeiGene, FDA, IND, MIBC, regulatory, Tislelizumab, US / June 15, 2021 June 15, 2021

Shots: The US FDA has approved the IND application of oral Asieris’ APL-1202 + BeiGene’s tislelizumab as neoadjuvant therapy in patients with MIBC The P-I/II study will evaluate the safety of the dual regimen in MIBC patients, determine the RP2D and assess the efficacy of combo as neoadjuvant therapy for MIBC. Asieris will accelerate the …

BeiGene’s Tislelizumab in Combination with Asieris’ APL-1202 Receive the US FDA’s IND Approval for MIBC Read More »

Gilead’s Epclusa (Sofosbuvir/Velpatasvir) Receives the US FDA’s Approval for Children with Chronic Hepatitis C Infection

approval, Chronic Hepatitis C Infection, Epclusa, FDA, Gilead, regulatory, Sofosbuvir, US, Velpatasvir / June 11, 2021 June 11, 2021

Shots: The US FDA has approved the NDA of an oral pellet formulation of Epclusa (sofosbuvir 200 mg/velpatasvir 50 mg and sofosbuvir 150 mg/velpatasvir 37.5 mg) for children aged 3 yrs. who cannot swallow tablets The approval is based on a P-II trial evaluating Epclusa in 41 children with HCV for 12 wks. The study …

Gilead’s Epclusa (Sofosbuvir/Velpatasvir) Receives the US FDA’s Approval for Children with Chronic Hepatitis C Infection Read More »

Vertex’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) Receives the US FDA’s Approval in Children Aged 6 to 11 with Certain Mutations in Cystic Fibrosis

approval, Cystic fibrosis, elexacaftor, FDA, Ivacaftor, regulatory, tezacaftor, Trikafta, US, Vertex / June 10, 2021 June 10, 2021

Shots: The approval is based on a P-III study evaluating the safety, PK & efficacy of Trikafta in 66 children aged 6-11yrs. old with CF with either two copies of F508del mutations or one copy of F508del mutation & one minimal function mutation for 24wks. The company has filed the application in EMA & MHRA …

Vertex’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) Receives the US FDA’s Approval in Children Aged 6 to 11 with Certain Mutations in Cystic Fibrosis Read More »

Double resignation over Aduhelm approval depletes FDA’s CNS adcomm

aducanumab, Aduhelm, Alzheimer's, amyloid, Biogen, central nervous system, FDA, Market Access, neurology, News, regulatory / June 10, 2021 June 10, 2021

Two members of an FDA advisory committee have now resigned in protest over Monday’s approval of Biogen’s Alzheimer’s disease drug Aduhelm despite an overwhelming vote against doing so. Washington University neurologist Joel Perlmutter was the first to announce he was stepping down from the Peripheral and Central Nervous System Drugs Advisory Committee on the day …

Double resignation over Aduhelm approval depletes FDA’s CNS adcomm Read More »

Pfizer’s Prevnar 20 (Pneumococcal 20-Valent Conjugate Vaccine) Receives the US FDA’s Approval for the Prevention of Invasive Disease and Pneumonia

approval, Conjugate Vaccine, FDA, Invasive Disease, Pfizer, Pneumococcal 20-valent, pneumonia, PREVNAR 20, regulatory, US / June 9, 2021 June 9, 2021

Shots: The approval is based in the clinical program including P-I & II trials, and three P-III trials evaluating the safety & immunogenicity of Prevnar 20 in adults aged ≥18yrs. with invasive disease and pneumonia caused by the 20 S. pneumoniae serotypes This is the first approval of a vaccine that protects against 20 serotypes …

Pfizer’s Prevnar 20 (Pneumococcal 20-Valent Conjugate Vaccine) Receives the US FDA’s Approval for the Prevention of Invasive Disease and Pneumonia Read More »

AbbVie’s Rinvoq (upadacitinib) Receives the Health Canada’s Approval for the Treatment of Psoriatic Arthritis

AbbVie, approval, Health Canada, Psoriatic Arthritis, regulatory, Rinvoq, Upadacitinib / June 8, 2021 June 8, 2021

Shots: The approval is based on data from two P-III studies i.e SELECT-PsA 1 & 2 evaluating the safety & efficacy of Rinvoq vs PBO in 2,000+ patients with active PsA who have had an inadequate response to methotrexate or other DMARDs The studies met their 1EPs of ACR20 response @12wks., Rinvoq (15mg) showed non-inferiority …

AbbVie’s Rinvoq (upadacitinib) Receives the Health Canada’s Approval for the Treatment of Psoriatic Arthritis Read More »

BeiGene Reports NMPA’s Acceptance of sBLA for Tislelizumab to Treat MSI-H or dMMR Solid Tumors

Acceptance, BeiGene, dMMR, Microsatellite Instability-High, Mismatch Repair Deficient, MSI-H, regulatory, sBLA, Solid Tumors, Tislelizumab / June 8, 2021 June 8, 2021

Shots: The sBLA submission is based on results from a P-II study evaluating the efficacy & safety of tislelizumab (200 mg, IV, q3w) as monothx in 80 patients with previously treated LA, unresectable or metastatic MSI-H or dMMR solid tumors The results demonstrated that the therapy treatment was consistent & durable across tumor types and …

BeiGene Reports NMPA’s Acceptance of sBLA for Tislelizumab to Treat MSI-H or dMMR Solid Tumors Read More »

Roche’s SARS-CoV-2 Antigen Test Receives CE mark For Self Testing of COVID-19 at Home

CE Mark, COVID-19, MedTech, regulatory, Roche, SARS-CoV-2 Antigen Test / June 8, 2021 June 8, 2021

Shots: Roche received a CE mark for its SARS-CoV-2 Ag self-test nasal at home setting for COVID 19. The test will be available in countries accepting the CE mark The early version of the test is available in the EU under special approval pathways since Feb, 2021. The test can be performed by individuals using …

Roche’s SARS-CoV-2 Antigen Test Receives CE mark For Self Testing of COVID-19 at Home Read More »

Biogen and Eisai’s Aduhelm (aducanumab-avwa) Receive the US FDA’s Accelerated Approval for Alzheimer Disease

Accelerated Approval, aducanumab-avwa, Aduhelm, Alzheimer Disease, Biogen, Eisai, FDA, regulatory, US / June 8, 2021 June 8, 2021

Shots: The approval is based on the two P-III clinical trials i.e EMERGE and ENGAGE along with P-Ib PRIME study involves assessing the efficacy of Aduhelm vs PBO in patients with early stages of AD with confirmed presence of amyloid pathology Results: the therapy consistently showed a dose and time-dependent effect on the reduction of …

Biogen and Eisai’s Aduhelm (aducanumab-avwa) Receive the US FDA’s Accelerated Approval for Alzheimer Disease Read More »

Novo Nordisk’s Wegovy (semaglutide, 2.4mg) Receives the US FDA’s Approval for Weight Management in Adults with Obesity

approval, FDA, Novo Nordisk, obesity, regulatory, semaglutide, US, Wegovy, weight management / June 7, 2021 June 7, 2021

Shots: The approval is based on the results from the P-IIIa STEP clinical trial program evaluating Wegovy (2.4mg injection, qw) in ~4,500 adults with obesity or overweight with at least one weight-related comorbidity Results: the therapy showed 17-18% average weight loss in people without T2D @68wks. and has a safe and well-tolerated profile across the …

Novo Nordisk’s Wegovy (semaglutide, 2.4mg) Receives the US FDA’s Approval for Weight Management in Adults with Obesity Read More »

ProMetic’ Ryplazim (plasminogen) Receives the US FDA’s Approval as First Treatment for Patients with Plasminogen Deficiency

regulatory / June 7, 2021 June 7, 2021

Shots: The approval is based on the study assessing the effectiveness and safety of Ryplazim (every two to four days for 48 wks.) in 15 adult and pediatric patients with plasminogen deficiency type 1 The therapy showed at least 50% improvement in lesions in 11 patients with lesions at baseline and absence of recurrent or …

ProMetic’ Ryplazim (plasminogen) Receives the US FDA’s Approval as First Treatment for Patients with Plasminogen Deficiency Read More »

Dermavant Submits NDA to FDA for Tapinarof Cream to Treat Plaque Psoriasis

Dermavant, FDA, NDA, Plaque Psoriasis, regulatory, Tapinarof Cream / June 4, 2021 June 4, 2021

Shots: The submission is based on data from the P-III PSOARING 1 & PSOARING as well as interim results from PSOARING 3 long-term safety study 2 evaluating the safety and efficacy of tapinarof vs vehicle in patients with plaque psoriasis Results: Improvement in PGA scores of 0 or 1 with a minimum 2-grade improvement from …

Dermavant Submits NDA to FDA for Tapinarof Cream to Treat Plaque Psoriasis Read More »

Innovent and Lilly’s Tyvyt (sintilimab injection) + Gemcitabine and Platinum CT Receive NMPA’s Approval as 1L Therapy for Squamous Non-Small Cell Lung Cancer

approval, Gemcitabine, Innovent, Lilly, NMPA, Platinum CT, regulatory, Sintilimab injection, Squamous Non-Small Cell Lung Cancer, Tyvyt / June 4, 2021 June 4, 2021

Shots: The approval is based on P-III ORIENT-12 trial evaluating Tyvyt (sintilimab injection) or PBO + Gemzar (gemcitabine) & platinum CT in 357 patients in a ratio (1:1) as 1L therapy for advanced or metastatic sqNSCLC Results: Improvement in PFS & OS, m-PFS (5.5 vs 4.9 mos.) as assessed by IRRC & (6.7 vs 4.9 …

Innovent and Lilly’s Tyvyt (sintilimab injection) + Gemcitabine and Platinum CT Receive NMPA’s Approval as 1L Therapy for Squamous Non-Small Cell Lung Cancer Read More »

BMS’ Opdivo (nivolumab) + Yervoy (ipilimumab) Receive EC’s Approval for 1L Treatment for Unresectable Malignant Pleural Mesothelioma

regulatory / June 3, 2021 June 3, 2021

Shots: The approval is based on P-III CheckMate -743 study assessing Opdivo (3 mg/kg, q2w) + Yervoy (1 mg/kg, q6w) vs CT in patients with previously untreated MPM The trial met its 1EP i.e improvement in OS (26% reduction in risk of death) m-OS (18.1 vs 14.1 mos.), ORR (40% vs 43%), DoR (11.0 vs …

BMS’ Opdivo (nivolumab) + Yervoy (ipilimumab) Receive EC’s Approval for 1L Treatment for Unresectable Malignant Pleural Mesothelioma Read More »

Janssen’s Teclistamab Receives the US FDA’s Breakthrough Therapy Designation for Relapsed or Refractory Multiple Myeloma

Breakthrough Therapy Designation, FDA, Janssen, multiple myeloma, Refractory, regulatory, Relapsed, Teclistamab, US / June 2, 2021 June 2, 2021

Shots: The FDA’s BT and EMA’s PRIME designations are based on data from the P-I MajesTEC-1 study evaluating the safety and efficacy of teclistamab (Ab targeting BCMA and CD3) in adults with r/rMM. The findings will be presented at ASCO 2021 In preclinical studies, the therapy kills myeloma cell lines and bone marrow-derived myeloma cells …

Janssen’s Teclistamab Receives the US FDA’s Breakthrough Therapy Designation for Relapsed or Refractory Multiple Myeloma Read More »

Novartis’ Cosentyx (secukinumab) Receives the US FDA’s Approval for the Treatment of Moderate to Severe Plaque Psoriasis

Cosentyx, FDA, Moderate, Novartis, Plaque Psoriasis, regulatory, secukinumab, Severe, US / June 2, 2021 June 2, 2021

Shots: The approval is based on two P-III studies evaluating Cosentyx (75 mg/ 150 mg) vs PBO in children aged 6 to <18 yrs with plaque psoriasis The first study demonstrates reduction in psoriasis severity @12 wks., PASI 75 response for 75mg and 150mg (55%, 86% vs 10%, 19%), achieved clear or almost clear skin …

Novartis’ Cosentyx (secukinumab) Receives the US FDA’s Approval for the Treatment of Moderate to Severe Plaque Psoriasis Read More »

Biohaven’s Nurtec ODT (rimegepant) Receives the US FDA’s Approval for Treatment of Migraine

regulatory / June 1, 2021 June 1, 2021

Shots: The approval is based on the P-III OLE trial involves assessing Nurtec ODT (rimegepant, 75 mg) vs PBO in patients with episodic migraine who experience less than 15 headache days/mos. The 1EPs & 2EPs of the trial demonstrate a reduction in monthly migraine days by 4.3 days/mos. after 3mos. treatment, 50% patients reported at …

Biohaven’s Nurtec ODT (rimegepant) Receives the US FDA’s Approval for Treatment of Migraine Read More »

BMS’ Abecma (idecabtagene vicleucel) Receives the Health Canada’s Approval as the First Anti-BCMA CAR T Cell Therapy for Relapsed and Refractory Multiple Myeloma

Abecma, Anti-BCMA, approval, BMS, CAR T-cell Therapy, Health Canada, idecabtagene vicleucel, Refractory, regulatory, Relapsed / June 1, 2021 June 1, 2021

Shots: The conditional approval is based on the results of the P-II KarMMa study involves assessing Abecma in 122 patients with RRMM prior treated with 3L+ therapy including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 Ab and who are refractory to their last treatment Results: @median follow-up of 13.3 mos., ORR (74%); CR …

BMS’ Abecma (idecabtagene vicleucel) Receives the Health Canada’s Approval as the First Anti-BCMA CAR T Cell Therapy for Relapsed and Refractory Multiple Myeloma Read More »

Ipsen Reports the US FDA’s Acceptance of NDA for Palovarotene to Treat Fibrodysplasia Ossificans Progressiva

FDA, Fibrodysplasia Ossificans Progressiva, FOP, Ipsen, NDA, Palovarotene, regulatory, US / May 31, 2021 May 31, 2021

Shots: The NDA submission is based on data from the ongoing P-III MOVE trial evaluating the efficacy and safety of palovarotene (chronic/flare-up dosing regimen) in patients with FOP The post hoc analyses of 1EPs from the P-III MOVE trial demonstrated a 62% reduction in mean annualized new HO volume in patients with palovarotene vs untreated …

Ipsen Reports the US FDA’s Acceptance of NDA for Palovarotene to Treat Fibrodysplasia Ossificans Progressiva Read More »

Novo Nordisk Resubmits Label Expansion Application to the US FDA for Semaglutide (2.0mg)

FDA, label Expansion Application, Novo Nordisk, regulatory, Resubmits, semaglutide, US / May 31, 2021 May 31, 2021

Shots: The company has announced the resubmission of a label expansion application to the US FDA for the existing marketing authorization for Ozempic to introduce a new dose of 2.0mg for the Treatment of T2D The resubmission follows the Refusal to File letter received by the FDA on 22 March 2021. The standard review time …

Novo Nordisk Resubmits Label Expansion Application to the US FDA for Semaglutide (2.0mg) Read More »

BridgeBio and Helsinn’s Truseltiq (infigratinib) Receive the US FDA’s Approval for Cholangiocarcinoma

approval, BridgeBio, CCA, Cholangiocarcinoma, FDA, Helsinn, Infigratinib, regulatory, Truseltiq, US / May 31, 2021 May 31, 2021

Shots: The approval is based on a P-II study assessing Truseltiq (125mg, qd, for 21 days of 28-day cycles) in 108 patients who had undergone at least one prior treatment for advanced CCA. Out of 108, 107 had Stage IV CCA Results: ORR (23%); mDOR (5mos.); presented at ASCO 2021. Additional marketing applications for Truseltiq …

BridgeBio and Helsinn’s Truseltiq (infigratinib) Receive the US FDA’s Approval for Cholangiocarcinoma Read More »