FDA

Better late than never? Eczema drug now approved, Incyte says warning unwarranted

Atopic dermatitis patients now have a new option that comes as a topical cream. The FDA approved Incyte’s drug, Opzelura, as a treatment for the inflammatory skin condition, but the regulatory nod comes with a black box warning on the label that broadly covers the entire class of JAK inhibitors.

Incyte leads JAK push into eczema as FDA clears Opzelura cream

The FDA has approved Incyte’s Opzelura cream for atopic dermatitis, commonly called eczema, becoming the first and only topical JAK inhibitor registered in the US. Opzelura (ruxolitinib) – which has been cleared to treat atopic dermatitis in adolescents and adults whose symptoms cannot be treated using other topical therapies – has beaten its orally-administered rivals …

Incyte leads JAK push into eczema as FDA clears Opzelura cream Read More »

Public Health Experts ‘Flabbergasted’ That Biden Still Hasn’t Picked an FDA Chief

President Joe Biden’s failure to name someone to lead the Food and Drug Administration, more than 10 months after the election, has flummoxed public health experts who say it’s baffling for the agency to be without a permanent leader during a national health crisis. The pandemic has taxed the FDA, an 18,000-person agency whose chiefs …

Public Health Experts ‘Flabbergasted’ That Biden Still Hasn’t Picked an FDA Chief 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

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

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 »

Seagen, Genmab antibody drug conjugate gets FDA approval in cervical cancer

The Seagen and Genmab antibody drug conjugate (ADC), named Tivdak, is approved to treat cases of cervical cancer that have returned or spread following treatment with chemotherapy. Tivdak is Seagen’s third ADC; for Genmab, the drug will become its first commercialized product.

FDA clears Seagen, Genmab’s Tivdak ADC for cervical cancer

Antibody-drug conjugate (ADC) specialist Seagen has claimed its fourth product approval in the US, getting the nod from the FDA for Tivdak as a second-line monotherapy for recurrent or metastatic cervical cancer. The accelerated approval for Tivdak (tisotumab vedotin) is for women whose cancer has progressed on or after first-line chemotherapy, according to the two …

FDA clears Seagen, Genmab’s Tivdak ADC for cervical cancer Read More »

Pfizer, BioNTech prep low-dose COVID jab filing for kids aged 5 to 11

With positive phase 2/3 results for their COVID-19 vaccine Comirnaty in hand for the five to 11 age group, Pfizer and BioNTech are planning to move swiftly ahead with a filing for emergency use that could be approved in a matter of weeks. The data is the first generated with any COVID-19 vaccine in this …

Pfizer, BioNTech prep low-dose COVID jab filing for kids aged 5 to 11 Read More »

Pfizer, BioNTech prep low-dose COVID jab filing for kids aged 5 to 11

With positive phase 2/3 results for their COVID-19 vaccine Comirnaty in hand for the five to 11 age group, Pfizer and BioNTech are planning to move swiftly ahead with a filing for emergency use that could be approved in a matter of weeks. The data is the first generated with any COVID-19 vaccine in this …

Pfizer, BioNTech prep low-dose COVID jab filing for kids aged 5 to 11 Read More »

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

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 »

Biogen and Samsung land FDA approval for biosimilar to blockbuster Roche drug

The FDA has approved the first biosimilar that references the blockbuster Roche drug Lucentis. The Biogen and Samsung BioLogics joint venture that developed the biologic product have approval to treat three eye conditions that lead to vision loss.

Pfizer reports positive Covid-19 vaccine data in kids; FDA is filing coming

A lower-dose version of Pfizer and BioNTech’s Covid-19 vaccine showed safety and efficacy results in children 5 to 11 that were comparable to those in an older age group. With these preliminary data, the companies said they plan to seek emergency use authorization of the shots.

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

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 »

FDA panel backs booster shots in high-risk groups after rejecting broader proposal

An FDA advisory panel voted down a proposal for booster shots of the Pfizer/BioNTech Covid-19 in the general population, opting instead to support third shots in certain high-risk groups, such as the elderly. Panelists want to see more information supporting the need for a booster, as well as data about a heart safety risk the …

FDA panel backs booster shots in high-risk groups after rejecting broader proposal Read More »

Takeda therapy gets speedy FDA nod for lung cancers with rare genetic signature

Takeda Pharmaceutical drug mobocertinib was approved by the FDA to treat non-small cell lung cancer in patients whose tumors carry a rare genetic signature. With the approval, Takeda can offer an oral alternative to a recently approved Johnson & Johnson drug that targets the same mutation but is given as an infusion.

Lead Contamination in Calcium Supplements 

Do calcium citrate and calcium carbonate have as much lead as calcium supplements derived from dolomite and animal bone?  You may remember I’ve explored the role calcium supplements may play in reducing lead exposure during pregnancy and breastfeeding, but that assumes the supplements themselves are not contaminated. Supplements with lead?  It all started with an extraordinary case report published in 1977, entitled “Lead-Contaminated Health Food.” As I discuss in my video Lead in Calcium …

Lead Contamination in Calcium Supplements  Read More »

Takeda gets a win for its Wave1 pipeline, as Exkivity nabs FDA approval

Takeda has had a run of bad luck with its Wave1 pipeline of new drug candidates of late, but can now celebrate a vestry after getting FDA approval for first-in-class lung cancer therapy Exkivity. The US regulator has cleared the tyrosine kinase inhibitor (TKI) as a second-line treatment for locally advanced or metastatic non-small-cell lung …

Takeda gets a win for its Wave1 pipeline, as Exkivity nabs FDA approval Read More »

Ahead of FDA adcomm, Pfizer, Moderna say COVID jab efficacy dips

Pfizer and Moderna have both reported data that they say backs up the need for booster shots of their COVID-19 vaccines ahead of an FDA advisory committee meeting tomorrow, although the regulator seems less convinced. Documents published ahead of the panel by the FDA reviewers suggest that there is some evidence from observational studies that …

Ahead of FDA adcomm, Pfizer, Moderna say COVID jab efficacy dips Read More »

How Fauci and the NIH Got Ahead of the FDA and CDC in Backing Boosters

In January — long before the first jabs of covid-19 vaccine were even available to most Americans — scientists working under Dr. Anthony Fauci at the National Institute of Allergy and Infectious Diseases were already thinking about potential booster shots. A month later, they organized an international group of epidemiologists, virologists and biostatisticians to track …

How Fauci and the NIH Got Ahead of the FDA and CDC in Backing Boosters Read More »

Takeda’s Exkivity (mobocertinib) Receives the US FDA’s Approval as the First Oral Therapy for EGFR Exon20 Insertion+ NSCLC

Shots: The approval is based on the P-I/II trial evaluating Exkivity (160 mg) in 114 patients with EGFR Exon20 insertion+ NSCLC who received prior Pt-based therapy The results demonstrated ORR (28%) as assessed by IRC & 35% as per investigator, m-DoR (17.5mos.), m-OS (24mos.), and m-PFS (7.3mos.) as per IRC Exkivity is a TKI inhibitor …

Takeda’s Exkivity (mobocertinib) Receives the US FDA’s Approval as the First Oral Therapy for EGFR Exon20 Insertion+ NSCLC Read More »

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

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 »

Astellas Pharma reports fourth patient death in gene therapy clinical trial

The death occurred less than two weeks after Astellas Pharma reported that the patient developed liver problems after being dosed with the experimental gene therapy for a rare neuromuscular disorder. Astellas said the cause of death is still under investigation, and the FDA has placed a clinical hold on the study.

First FDA filing for Novartis’ latecomer PD-1 drug tislelizumab

Novartis has filed for FDA approval of tislelizumab – a PD-1 inhibitor licensed from Chinese biotech BeiGene earlier this year – as a second-line treatment for oesophageal cancer. The Swiss pharma giant licensed ex-China rights to tislelizumab in February for $650 million upfront ad up to $1.55 billion in milestones, shortly after its own PD-1 …

First FDA filing for Novartis’ latecomer PD-1 drug tislelizumab Read More »

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

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 »

FDA sets expectations for pediatric Covid-19 vaccines; decision could come mid-fall

Acknowledging the demand for a Covid-19 vaccine for children, the FDA said it expects regulatory review could take weeks, not months, depending on the timing of a submission from vaccine developers. U.S. health officials told Reuters they expect the Pfizer/BioNTech vaccine will be the first one ready for an FDA submission.

FDA warns Kaleido Bio to test its Covid-19 candidate as a drug, not a medical food

The FDA issued a warning letter to Kaleido Biosciences alleging the company tested its experimental microbiome product in Covid-19 patients without submitting a formal clinical trial application. Kaleido claims the filing was not required because the product is a medical food, but the regulator doesn’t buy that argument.

Cancer concerns lead FDA to place clinical hold on BioMarin gene therapy

A Phase 1/2 study of BioMarin Pharmaceutical’s gene therapy for phenylketonuria has been placed under a clinical hold after interim results from a preclinical study showed that some mice developed liver tumors. Patients did not receive the same high dose that was given to the mice, and no one in the clinical trial has developed …

Cancer concerns lead FDA to place clinical hold on BioMarin gene therapy Read More »

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

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 »

FDA slaps clinical hold on BioMarin’s PKU gene therapy

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal. The biotech said that the US regulator ordered the pause on the study after liver tumours were …

FDA slaps clinical hold on BioMarin’s PKU gene therapy Read More »

BioMarin Reports FDA’s Hold on P-I/II Phearless Trial of BMN 307 in Adults with PKU

Shots: The FDA’s clinical hold is based on interim safety findings from a pre-clinical study which showed that 6/7 animals treated with BMN 307 (2e14 Vg/kg) had tumors on liver necropsy @52wks. after dosing with evidence for integration of portions of AAV vector into the genome No lesions were observed in any mice @24wks. 5 …

BioMarin Reports FDA’s Hold on P-I/II Phearless Trial of BMN 307 in Adults with PKU Read More »

Impel NeuroPharma’s drug-device combo product nabs FDA nod for migraine

The FDA improved an Impel NeuroPharma migraine treatment, a nasal spray intended to bring pain relief more quickly than drugs administered in other ways. It marks the first approval for the Seattle company, which has developed a proprietary nasal delivery technology.

The impact of monoclonal antibodies and COVID-19 recovery

As the United States and world continue to fight the COVID-19 pandemic, America’s biopharmaceutical research companies continue to do our part in fighting this deadly virus. Decades of scientific innovation have allowed biopharmaceutical researchers to respond quickly and continue to research and develop treatments and vaccines for COVID-19. Importantly, the best strategy to prevent infection …

The impact of monoclonal antibodies and COVID-19 recovery Read More »

Assembly Bio drops hepatitis B drug after liver problems surface in clinical trial

Four patients dosed with an experimental Assembly Biosciences hepatitis B treatment developed high levels of liver enzymes, a sign of drug-induced liver toxicity. The biotech has decided to stop further work on that drug and focus instead on developing its other clinical-stage hepatitis B drugs.

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

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 »

FDA schedules adcomm for Pfizer’s COVID booster filing

The FDA has said it will hold an advisory committee meeting later this month to discuss Pfizer and BioNTech’s filing for approval of a third booster dose of their COVID-19 vaccine Comirnaty in people aged 16 and over. The adcomm is scheduled for 17 September and will discuss the Pfizer/BioNTech filing as well as booster …

FDA schedules adcomm for Pfizer’s COVID booster filing Read More »

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

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 »

FDA firms up JAK inhibitor warnings after Xeljanz review

The FDA has concluded its safety review of Pfizer’s JAK inhibitor Xeljanz and Xeljanz XR, requiring revised warnings for the drugs as well as others in the class after finding evidence of elevated risks of serious heart-related events. After looking at the data from Pfizer’s long-term, post-marketing safety study – ORAL Surveillance – the US …

FDA firms up JAK inhibitor warnings after Xeljanz review Read More »

FDA cracks down on JAK drug class, calls for new warnings of heart, cancer risks

The FDA’s review of a post-marketing study for Pfizer blockbuster anti-inflammatory drug Xeljanz found a higher risk of cardiovascular problems and cancer, and the agency is requiring additional warnings flagging those risks. The updated warnings extend to AbbVie and Eli Lilly medicines in the same drug class.

FDA clears J&J’s twice-yearly Invega for schizophrenia

One of the biggest challenges to treating schizophrenia is non-compliance with treatment, but Johnson & Johnson’s pharma unit Janssen is trumpeting a new version of its Invega drug that could solve that problem. The FDA has just approved Janssen’s Invega Hafyera (paliperidone), a twice-yearly injectable formulation that extends the company’s big-selling brand and offers the …

FDA clears J&J’s twice-yearly Invega for schizophrenia Read More »

FDA Clears World’s First and Only Smart Knee Implant

What You Should Know: – Zimmer Biomet Holdings, a global medical technology leader, and Canary Medical, a medical data company, announced U.S. Food and Drug Administration (FDA) De Novo classification grant and authorization to market the tibial extension for Persona IQ®, the world’s first and only smart knee cleared by the FDA for total knee …

FDA Clears World’s First and Only Smart Knee Implant Read More »

Pfizer eczema contender beats Dupixent in study, but JAK drug class is still in limbo

Pfizer has preliminary data showing its experimental drug for atopic dermatitis beat a blockbuster drug from Regeneron Pharmaceuticals in a head-to-head clinical trial. But the future of that drug and others might depend more on an FDA review of safety data for medicines in this class of so-called JAK inhibitors.

MicroTransponder Receives the US FDA’s Approval for Vivistim System as the First Vagus Nerve Stimulation Implant for Stroke Rehabilitation

Shots: The approval is based on a study of 108 patients in the US and the UK, that showed patients treated with the Vivistim system had improved mobility than patients in the control group The device used in conjunction with rehabilitation therapy to improve motor function in the hands & arms following ischemic stroke. The …

MicroTransponder Receives the US FDA’s Approval for Vivistim System as the First Vagus Nerve Stimulation Implant for Stroke Rehabilitation Read More »

Journalists Give Rundown on Vaccine Mandates and FDA’s Official Approval of the Pfizer Shot

KHN correspondent Rachana Pradhan discussed covid vaccine mandates and the Food and Drug Administration’s recent approval of the Pfizer-BioNTech vaccine on Newsy’s “Morning Rush” on Tuesday. Click here to watch Pradhan on “Morning Rush” KHN Midwest correspondent Cara Anthony talked about covid vaccines on Illinois Public Media’s “The 21st Show” on Monday. Click here to …

Journalists Give Rundown on Vaccine Mandates and FDA’s Official Approval of the Pfizer Shot Read More »

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

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 »

KHN’s ‘What the Health?’: Vaccine Approval Moves the Needle on Covid

Can’t see the audio player? Click here to listen on SoundCloud. You can also listen on Spotify, Apple Podcasts, Stitcher, Pocket Casts or wherever you listen to podcasts. The Food and Drug Administration gave full approval this week to the covid-19 vaccine from Pfizer and BioNTech, which will henceforth be known as “Comirnaty.” It is …

KHN’s ‘What the Health?’: Vaccine Approval Moves the Needle on Covid Read More »

ADC Therapeutics strikes a $325M royalty deal to fuel its cancer drug strategy

The royalty agreement with HealthCare Royalty Partners will support ADC Therapeutics’ plans to commercialize its newly approved cancer drug and develop the next one in its pipeline. The biotech’s drug Zynlonta is the first of its type approved by the FDA as a treatment for diffuse large cell B-cell lymphoma

The FDA’s Culture: Should Safety Dominate All Practices?

By STEVEN ZECOLA An organization’s culture is an internal set of shared values, attitudes and practices. The cohesiveness of the organizational culture will affect whether the entity will meet its vision, purpose, and goals. One type of organizational culture is hierarchical in nature.   Unlike a risk-taking culture, this structure features policy, process and precision. It is …

The FDA’s Culture: Should Safety Dominate All Practices? Read More »

Apple, Bose and Others Pump Up the Volume on Hearing Aid Options, Filling Void Left by FDA

Spurred by decades of complaints about the high cost of hearing aids, Congress passed a law in 2017 to allow over-the-counter sales, with hopes it would boost competition and lower prices. Four years later, federal regulators have yet to issue rules to implement the law. But changes in the industry are offering consumers relief. In …

Apple, Bose and Others Pump Up the Volume on Hearing Aid Options, Filling Void Left by FDA 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

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 »

Scant on details, J&J claims it has data supporting Covid-19 booster shots

As U.S. plans for Covid-19 booster shots unfold, Johnson & Johnson is angling to join the mix, reporting data that it says support a second shot of its vaccine. But the data the company points to are sparse and it’s not entirely clear they support booster shots.

FDA approves first drug for severe itching in chronic kidney disease patients

Chronic kidney disease patients who experience pruritus after dialysis can now get relief from the first FDA-approved treatment for the condition. Cara Therapeutics received the FDA’s nod green light for its injectable drug, Korsuva.

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

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 »

Pfizer, BioNTech Covid-19 vaccine is awarded full FDA approval

Full FDA approval makes the Pfizer/BioNTech Covid-19 vaccine the first to reach that milestone. But the regulatory decision also flagged a potential risk of heart inflammation, particularly in young males, and the agency has asked the companies to conduct additional clinical research.

FDA grants full approval to Pfizer/BioNTech COVID jab

Pfizer and BioNTech’s COVID-19 vaccine was the first to get emergency use authorisation (EUA) from the FDA, and has also become the first to get full regulatory approval in the US. The regulator has cleared the vaccine – now officially given the trade name Comirnaty – to prevent COVID-19 in people aged 16 or more, …

FDA grants full approval to Pfizer/BioNTech COVID jab Read More »

FDA issues warning on robot-assisted breast cancer surgery

The FDA has issued a safety alert about the use of robot-assisted surgical (RAS) devices for use in mastectomy procedures, pointing out that their safety and efficacy have not been established. The move comes amid reports that some people are being offered RAS for the prevention or treatment of breast cancer, sometimes as part of …

FDA issues warning on robot-assisted breast cancer surgery Read More »

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

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 »

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

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 »

HHS cites real-world data for Covid-19 boosters; WHO says the science is still out

In announcing plans to offer Covid-19 booster shots, federal health officials cited declining vaccine efficacy shown by real-world studies. But the World Health Organization said the science is less than clear about booster shots, adding that it’s ethically wrong to give third shots when so many around the world are waiting for their first one.

Jardiance gets parity with Farxiga in US as FDA clears use in heart failure

Eli Lilly and Boehringer Ingelheim have claimed FDA approval for their diabetes therapy Jardiance in heart failure with reduced ejection fraction (HFreF), setting up a market tussle with AstraZeneca’s Farxiga. The US regulator has cleared a 10mg daily dose of Jardiance (empagliflozin) to reduce the risk of cardiovascular death plus hospitalisation for heart failure in …

Jardiance gets parity with Farxiga in US as FDA clears use in heart failure Read More »

Lo que debes saber sobre el refuerzo de la vacuna contra covid

La administración de Biden anunció el miércoles 18 de agosto planes para ofrecer refuerzos de la vacuna contra covid-19 a todos los adultos estadounidenses a partir del próximo mes, y dijo que los datos recientes, incluidos algunos disponibles solo en los últimos días, jugaron un papel en esa decisión. “Si esperan a que suceda algo …

Lo que debes saber sobre el refuerzo de la vacuna contra covid Read More »

FDA decision for GSK drug opens door to treatment of more cancer patients

GlaxoSmithKline drug Jemperli is now FDA approved for treating solid tumors throughout the body as long as they have a particular genetic signature. Expanding the drug’s approval means the drug can treat more patients who have more types of cancer, and it also gives GSK a way to grow revenue in its thin oncology portfolio.

GSK claims broader ’tissue-agnostic’ label for PD-1 drug Jemperli

The FDA has approved GlaxoSmithKline’s latecomer PD-1 inhibitor Jemperli for a second use that will significantly extend the patient population eligible for treatment with the drug. Jemperli (dostarlimab) can now be used to treat adults with mismatch repair-deficient (dMMR) recurrent or advanced solid tumours – regardless of where they are located in the body – …

GSK claims broader ’tissue-agnostic’ label for PD-1 drug Jemperli Read More »

Rush to Boosters Sparks Confusion, Differing Recommendations

When the Food and Drug Administration announced last week that a third dose of Moderna or Pfizer-BioNTech covid-19 vaccine may boost the immunity of some people who are immunocompromised, officials repeated their stance that fully vaccinated, healthy people do not need another dose. With this caveat: “The FDA is actively engaged in a science-based, rigorous …

Rush to Boosters Sparks Confusion, Differing Recommendations Read More »

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

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 »

‘Tainted’ Blood: Covid Skeptics Request Blood Transfusions From Unvaccinated Donors

The nation’s roiling tensions over vaccination against covid-19 have spilled into an unexpected arena: lifesaving blood transfusions. With nearly 60% of the eligible U.S. population fully vaccinated, most of the nation’s blood supply is now coming from donors who have been inoculated, experts said. That’s led some patients who are skeptical of the shots to …

‘Tainted’ Blood: Covid Skeptics Request Blood Transfusions From Unvaccinated Donors 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

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 »

Ipsen hit by new palaver for rare disease drug palovarotene

Just as Ipsen’s $1 billion bet on rare bone disease drug palovarotene seemed to be nearing the finish line, it has fallen at the final hurdle. After a string of setbacks with the programme, Ipsen finally filed palovarotene with the FDA in May, earning a priority review. But the French drugmaker has already withdrawn it, …

Ipsen hit by new palaver for rare disease drug palovarotene Read More »

Merck’s Welireg becomes first drug for rare VHL tumours in US

Merck & Co has extended its range of cancer medicines in the US after claiming FDA approval for Welireg – a drug it acquired as part of its $2.2 billion takeover of Peloton Therapeutics in 2019 – for a family of rare tumours. The drug is part of Merck’s efforts to expand its oncology portfolio …

Merck’s Welireg becomes first drug for rare VHL tumours in US Read More »

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

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 drug acquired in $1B deal approved to treat tumors sparked by rare disease

FDA approval of the Merck drug, belzutifan, covers treatment of tumors caused by von Hippel-Lindau disease, an inherited disorder associated with several types of cancer. Merck added the small molecule to its pipeline two years ago as part of a $1.2 billion acquisition.

Mensaje del CEO de Pfizer al público: confíen en nosotros con el refuerzo de la vacuna contra covid

El CEO de Pfizer, Albert Bourla, confiaba en junio en la capacidad de la vacuna de su empresa para proteger contra la variante delta, altamente contagiosa, mientras se diseminaba por el mundo y llenaba de pacientes los hospitales estadounidenses. Apenas unas semanas después, Pfizer comunicó que solicitaría autorización para una vacuna de refuerzo, después que …

Mensaje del CEO de Pfizer al público: confíen en nosotros con el refuerzo de la vacuna contra covid Read More »

FDA authorizes Covid-19 booster shots, but only for the immunocompromised

Covid-19 vaccine booster shots are now authorized for organ transplant patients and others who have weakened immune systems. The FDA based its update on the results of two small studies that tested third shots of the mRNA Covid vaccines from Moderna and Pfizer/BioNTech.

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

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 »

Pfizer CEO to Public: Just Trust Us on the Covid Booster

Pfizer CEO Albert Bourla was confident in June about the ability of his company’s vaccine to protect against the highly contagious delta variant, as it marched across the globe and filled U.S. hospitals with patients. “I feel quite comfortable that we cover it,” Bourla said. Just weeks later, Pfizer said it would seek authorization for …

Pfizer CEO to Public: Just Trust Us on the Covid Booster Read More »

FDA authorises COVID booster jabs for at-risk people

The FDA has cleared the use of third dose of either the Pfizer/BioNTech or Moderna COVID-19 vaccines for people with weakened immune systems, but stopped short of a broader booster campaign. The emergency use authorisation (EUA) for the two mRNA-based vaccines has been amended so they can be used for solid organ transplant recipients or …

FDA authorises COVID booster jabs for at-risk people Read More »

Aprea Reports the FDA’s Hold on Clinical Trials for the Treatment Lymphoid Malignancy

Shots: The US FDA has put a partial clinical hold on Aprea’s clinical program that evaluates eprenetapopt + acalabrutinib or with venetoclax and rituximab in patients with lymphoid malignancies. The FDA’s decision is related to the safety & efficacy data from the P-III MDS study The patient enrollment will be pause until the clinical hold …

Aprea Reports the FDA’s Hold on Clinical Trials for the Treatment Lymphoid Malignancy Read More »

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

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 »

Second FDA clinical hold for Aprea cancer drug cites safety data in another study

For the second time in a week, the FDA placed a clinical hold on an Aprea Therapeutics cancer drug. The biotech said the agency cited safety and efficacy data from a prior failed clinical trial that tested the small molecule in a different form of cancer.

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

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

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 »

FDA rejects FibroGen/AZ’s roxadustat, asking for new trial

The FDA has demanded another clinical trial of FibroGen and AstraZeneca’s roxadustat for anaemia caused by chronic kidney disease (CKD) before it will consider approval, setting up a lengthy delay to the programme. The need for a new trial is something of a worst-case scenario for roxadustat, although a rejection was expected after an FDA …

FDA rejects FibroGen/AZ’s roxadustat, asking for new trial Read More »

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

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 »

Boston’s EXALT Model B Single-Use Bronchoscope Receives the US FDA’s Clearance for Bronchoscopy Procedures

Shots: The US FDA has granted 510(k) clearance for EXALT Model B single-use Bronchoscope to use in bedside procedures within ICU and OR and will be available in the US in the coming wks. The device is being used for bronchoscopy procedures including secretion management, airway intubation, percutaneous tracheostomy, double-lumen endotracheal tube placement, and biopsies …

Boston’s EXALT Model B Single-Use Bronchoscope Receives the US FDA’s Clearance for Bronchoscopy Procedures Read More »

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

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 »

Axsome faces delay for depression drug, hitting shares hard

The FDA was supposed to be delivering its verdict on Axsome Therapeutics’ depression therapy AXS-05 in less than a fortnight – but instead has delivered the company a letter outlining deficiencies in its marketing application. The problem for the company is that it doesn’t yet know what those deficiencies are, and that uncertainty weighed heavily …

Axsome faces delay for depression drug, hitting shares hard Read More »

Dermavant Reports the US FDA’s Acceptance of NDA for Tapinarof Cream to Treat Plaque Psoriasis

Shots: The NDA is based on P-III PSOARING 1 & 2 along with interim results from PSOARING 3 OLE study evaluating the safety & efficacy of tapinarof vs vehicle in patients with PsO In PSOARING 1 & 2 results: patients achieved a PGA score of 0 or 1 (35.4% vs 6% & 40.2% vs 6.3%) …

Dermavant Reports the US FDA’s Acceptance of NDA for Tapinarof Cream to Treat Plaque Psoriasis Read More »

FDA: Why we approved Prograf based on real-world data

Recently, the Food and Drug Administration (FDA) approved Prograf (tacrolimus) in combination with other immunosuppressant drugs for the new indication of preventing organ rejection for patients who have received a lung transplant. While the FDA approving a new indication isn’t typically news, this is a noteworthy event because (i) it marks the first approval of …

FDA: Why we approved Prograf based on real-world data Read More »

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

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

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 »

Sanofi extends its Pompe range with first Nexviazyme OK

Sanofi has added another string to its Pompe disease therapy bow, after getting FDA approval for Nexviazyme, its latest therapy for the rare, inherited disorder. The US regulator has approved Nexviazyme (avalglucosidase alfa) for the treatment of patients aged one year of age and older with late-onset Pompe disease, which progressively attacks the heart and …

Sanofi extends its Pompe range with first Nexviazyme OK Read More »

KHN’s ‘What the Health?’: Delta Blues

Can’t see the audio player? Click here to listen on SoundCloud. You can also listen on Spotify, Apple Podcasts, Stitcher, Pocket Casts or wherever you listen to podcasts. The U.S. is experiencing another surge of covid-19, particularly in Southern states where vaccination rates are generally lower than in other regions. But partisan fights rage on …

KHN’s ‘What the Health?’: Delta Blues Read More »

Lilly sets out its stall on donanemab as filing approaches

The FDA’s approval of Biogen’s Aduhelm for Alzheimer’s disease emboldened Eli Lilly to move forward plans to file its own candidate donanemab later this year, although it has acknowledged it will have to overcome reservations about anti-amyloid drugs. Lilly’s chief scientific officer Dan Skovronsky said on the company’s second-quarter results call that the results of …

Lilly sets out its stall on donanemab as filing approaches Read More »

Failed trial nixes another FDA approval, this time for BMS’ Istodax

Bristol-Myers Squibb’s HDAC inhibitor Istodax has been on the US market for a decade as a treatment for peripheral T-cell lymphoma (PTCL), but will now be withdrawn from sale after a failed phase 3 trial. Istodax (romidepsin) – originally developed by Celgene – is the latest in a string of cancer drugs originally given accelerated …

Failed trial nixes another FDA approval, this time for BMS’ Istodax Read More »

Novartis cleared to restart trials of new Zolgensma delivery route

The FDA has relaxed a clinical hold on clinical trials of Novartis’ intrathecal formulation of spinal muscular atrophy (SMA) gene therapy Zolgensma, allowing a new phase 3 trial to get underway. Zolgensma (onasemnogene abeparvovec) is already approved as an intravenous treatment in babies and very young children for the rare muscle-wasting disease, but the new …

Novartis cleared to restart trials of new Zolgensma delivery route Read More »

Abbott bags FDA okay for AI-based heart imaging software

The FDA has approved an artificial intelligence-based imaging software developed by Abbott that is used to help physicians carry out procedures like implanting stents to open up narrowed blood vessels in the heart.  Abbott‘s Ultreon software is billed as a first-of-its-kind platform that is designed to be used with optical coherence tomography (OCT), an imaging …

Abbott bags FDA okay for AI-based heart imaging software Read More »

PharmaShots Interview: Xencor’s Bassil I. Dahiyat Shares Insight on the US FDA’s Approval of COVID-19 Antibody Treatment Leveraging its Technology

In an interview with PharmaShots, Dr. Bassil I. Dahiyat, President and CEO of Xencor shared his views on the company’s collaborations with BMS and its Xtend XmAb technology. He also shed light on the EUA of Sotrovimab for COVID-19. Shots: The FDA has granted EUA to Vir and GSK’s sotrovimab, which was engineered with Xencor’s …

PharmaShots Interview: Xencor’s Bassil I. Dahiyat Shares Insight on the US FDA’s Approval of COVID-19 Antibody Treatment Leveraging its Technology Read More »

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

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 »

FDA gives breakthrough status to Biofourmis’ heart failure DTx

A digital therapeutic for patients with heart failure has been awarded breakthrough device status by the FDA – said by its developer Biofourmis  to be the first time the US regulator has given this designation for this type of product. Boston-based Biofourmis claimed the accolade for its BiovitalsHF prescription digital therapeutic (DTx), which is used …

FDA gives breakthrough status to Biofourmis’ heart failure DTx Read More »

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

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 »

FDA approves AZ’s anifromulab, ending 10-year lupus drug drought

AstraZeneca has completed the resurrection of its anifrolumab drug for systemic lupus erythematosus (SLE) after claiming FDA approval for the drug as an add-on therapy for adults with the autoimmune disease. Anifrolumab was all-but written off in 2018 after failing the phase 3 TULIP-1 trial, but bounced back with positive data from a second study …

FDA approves AZ’s anifromulab, ending 10-year lupus drug drought Read More »

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

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 »

Biofourmis Earns FDA Breakthrough Device Designation for Digital Therapeutic for Heart Failure

What You Should Know: – Biofourmis, a Boston-based global leader in virtual care and digital therapeutics, announced its BiovitalsHF® solution is the first-ever heart failure digital therapeutic to receive a Breakthrough Device designation from the U.S. Food and Drug Administration (FDA). – The Breakthrough Devices Program was created to provide patients and providers with timely …

Biofourmis Earns FDA Breakthrough Device Designation for Digital Therapeutic for Heart Failure Read More »

Medtronic Awarded FDA Clearance for 2 AI Algorithms for Cardiac Monitoring

What You Should Know: – Medtronic announced U.S. Food and Drug Administration (FDA) clearance for two AccuRhythm™ AI algorithms for use with the LINQ II™ insertable cardiac monitor (ICM) – and the presentation of AccuRhythm AI validation data this week at Heart Rhythm 2021. – AccuRhythm AI applies artificial intelligence (AI) to heart rhythm event …

Medtronic Awarded FDA Clearance for 2 AI Algorithms for Cardiac Monitoring Read More »

Diabetes Drug’s New Weight Loss Formula Fuels Cost-Benefit Debate

The long list of side effects that follow ads for the newer expensive drugs to treat Type 2 diabetes sometimes include an unusual warning: They might cause weight loss. That side effect is one that many people — especially those with Type 2 diabetes, which is associated with obesity — may desperately want. So it’s …

Diabetes Drug’s New Weight Loss Formula Fuels Cost-Benefit Debate Read More »

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

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 »

FDA turns down Ardelyx’s chronic kidney disease drug and asks for another trial

Ardelyx drug tenapanor was rejected by the FDA as a treatment for a complication experienced by chronic kidney disease patients. The regulatory decision came two weeks after the company received an FDA letter citing deficiencies in the drug application.

PharmaShots Interview: Guardant Health’s Justin Odegaard Shares Insight on the US FDA’s Approval of Guardant360 CDx for Amgen’s Lumakras

In an interview with PharmaShots, Justin Odegaard, Vice President of Clinical Development at Guardant Health shares his views on the Guardant360 CDx approval as the first & only liquid biopsy companion diagnostic for Amgen’s Lumakras (sotorasib) KRASG12C inhibitor for use in advanced NSCLC. Shots: Guardant Health received the US FDA’s approval for Guardant360 CDx liquid …

PharmaShots Interview: Guardant Health’s Justin Odegaard Shares Insight on the US FDA’s Approval of Guardant360 CDx for Amgen’s Lumakras Read More »

Viatris and Biocon’s Semglee (biosimilar, insulin glargine) Receive the US FDA’s Approval as the First Interchangeable Biosimilar for Diabetes

Shots: The US FDA has approved Semglee (insulin glargine-yfgn injection) as the first interchangeable biosimilar product under the 351(k) regulatory pathways to treat diabetes Semglee will allow pharmacy level substitution for the reference product, Lantus across the US & will be launch before the end of 2021. Biocon is eligible to have exclusivity for 12mos. …

Viatris and Biocon’s Semglee (biosimilar, insulin glargine) Receive the US FDA’s Approval as the First Interchangeable Biosimilar for Diabetes Read More »

FDA okays Mylan insulin drug as first ‘interchangeable’ biosimilar

Generic drugmaker Mylan has become the first company to secure FDA approval for a biosimilar product that is considered completely interchangeable with the reference product – namely Sanofi’s once-daily insulin Lantus. Mylan’s Semglee has been approved for over a year as a regular biosimilar to Lantus (insulin glargine), meaning that it could be used in …

FDA okays Mylan insulin drug as first ‘interchangeable’ biosimilar Read More »

Pfizer Court Fight Could Legalize Medicare Copays and Unleash ‘Gold Rush’ in Sales

Three years ago, pharma giant Pfizer paid $24 million to settle federal allegations that it was paying kickbacks and inflating sales by reimbursing Medicare patients for out-of-pocket medication costs. By making prohibitively expensive medicine essentially free for patients, the company induced them to use Pfizer drugs even as the price of one of those medicines, …

Pfizer Court Fight Could Legalize Medicare Copays and Unleash ‘Gold Rush’ in Sales Read More »

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

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 »

After false start, Merck claims Keytruda approval in early breast cancer

Merck & Co’s Keytruda has just picked up its 30th approval from the FDA, and it is a good one – early-stage triple-negative breast cancer (TNBC), a particularly hard-to-treat form of the disease. The FDA has given a green light to the use of Keytruda (pembrolizumab) pre-surgery alongside chemotherapy in the neoadjuvant setting, as well …

After false start, Merck claims Keytruda approval in early breast cancer Read More »

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

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 »

Bristol Myers pulls Opdivo for use in liver cancer amid broader FDA scrutiny of speedy approvals

Bristol Myers Squibb blockbuster drug Opdivo is being pulled from the market after failing to meet the main goal of a confirmatory study. The voluntary withdrawal comes amid broader FDA scrutiny of cancer drugs that were given accelerated approval.

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

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 »

BMS pulls speedy Opdivo approval for liver cancer in US

Bristol-Myers Squibb has withdrawn its US marketing approval for PD-1 inhibitor Opdivo as a second-line, single-agent treatment for hepatocellular carcinoma, a common form of liver cancer, in people previously treated with Bayer/Onyx’s Nexavar. Opdivo (nivolumab) was awarded an accelerated approval in 2017 for second-line HCC treatment on the strength of mid-stage data, but failed to …

BMS pulls speedy Opdivo approval for liver cancer in US Read More »

Facing Headwinds on New Alzheimer’s Drug, Biogen Launches Controversial Campaign

Do you sometimes lose your train of thought or feel a bit more anxious than is typical for you? Those are two of the six questions in a quiz on a website co-sponsored by the makers of Aduhelm, a controversial new Alzheimer’s drug. But even when all responses to the frequency of those experiences are …

Facing Headwinds on New Alzheimer’s Drug, Biogen Launches Controversial Campaign Read More »

FDA nod for Albireo drug makes it the first for children with rare liver disorder

The FDA has approved Bylvay, an Albireo Pharma drug that treats the severe itching experienced by patients with progressive familial intrahepatic cholestasis (PFIC). Prior to this approval, the only treatment options for the rare liver disorder were surgical.

Novartis eyes New Year verdict from FDA on delayed Leqvio

Novartis has revealed that the FDA has accepted its resubmission for cholesterol-lowering drug Leqvio – rejected unexpectedly by the US regulator last year – and has set a new action date of 1 January 2022. The drugmaker refiled its application for Leqvio (inclisiran) earlier this month, trying to get the programme back on track in …

Novartis eyes New Year verdict from FDA on delayed Leqvio Read More »

Analysis: Necessary or Not, Covid Booster Shots Are Probably on the Horizon

The drugmaker Pfizer recently announced that vaccinated people are likely to need a booster shot to be effectively protected against new variants of covid-19 and that the company would apply for Food and Drug Administration emergency use authorization for the shot. Top government health officials immediately and emphatically announced that the booster isn’t needed right …

Analysis: Necessary or Not, Covid Booster Shots Are Probably on the Horizon Read More »

Hard times at Ardelyx as FDA rejects kidney disease drug tenapanor

The FDA has taken a longer than usual look at Ardelyx’ regulatory submission for tenapanor, a drug for high blood phosphate levels associated with chronic kidney disease (CKD), and found it wanting – sending the biotech’s shares into a tailspin. The US regulator had extended its review of the drug by three months – setting …

Hard times at Ardelyx as FDA rejects kidney disease drug tenapanor Read More »

ViewPoints Interview: Myovant’s Dave Marek Shares Insights on the US FDA’s Approval of Myfembree in Uterine Fibroids

In an interview with PharmaShots, Dave Marek. CEO of Myovant shares insights on the US FDA’s approval of Myfembree (relugolix 40mg, estradiol 1mg, and norethindrone acetate 0.5mg) in Uterine Fibroids. Shots: The US FDA has approved Myovant & Pfizer’s Myfembree (relugolix 40 mg, estradiol 1 mg, and norethindrone acetate 0.5 mg) as the first once-daily …

ViewPoints Interview: Myovant’s Dave Marek Shares Insights on the US FDA’s Approval of Myfembree in Uterine Fibroids Read More »

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

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 »

Cytokinetics stock soars on heart drug data that sets stage for Phase 3 studies

A Cytokinetics drug in development for treating a rare heart disorder has promising Phase 2 data suggesting it could compete against a Bristol Myers Squibb drug currently under FDA review. Cytokinetics plans to advance its small molecule to Phase 3 testing by the end of this year.

FDA okays Merck’s would-be Prevnar rival Vaxneuvance

The FDA has approved a new pneumococcal vaccine from Merck & Co, just over a month after Pfizer got a green light for a new version of its market leading Prevnar shot. Merck’s Vaxneuvance – formerly known as V114 – is the first major competition to Pfizer’s franchise in the big market for conjugate pneumococcal …

FDA okays Merck’s would-be Prevnar rival Vaxneuvance 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

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 »

FDA adcomm turns down FibroGen, AZ’s roxadustat for CKD anaemia

Safety concerns seem to have scuppered any hope of a near-term approval for FibroGen and AstraZeneca’s roxadustat for anaemia associated with chronic kidney disease (CKD) in the US, after FDA advisors voted comprehensively against the drug yesterday. Just one of the 14-member panel thought that roxadustat should be approved for non-dialysis-dependent CKD patients, and only …

FDA adcomm turns down FibroGen, AZ’s roxadustat for CKD anaemia Read More »

KHN’s ‘What the Health?’: Here Comes Reconciliation

Can’t see the audio player? Click here to listen on SoundCloud. You can also listen on Spotify, Apple Podcasts, Stitcher, Pocket Casts or wherever you listen to podcasts. The expansion of health benefits is a major piece of the tentative budget deal reached this week by Democrats in Congress. They plan to press ahead — …

KHN’s ‘What the Health?’: Here Comes Reconciliation Read More »

FDA staffers defend Aduhelm approval in JAMA editorial

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 »

Dying Patients With Rare Diseases Struggle to Get Experimental Therapies

At 15, Autumn Fuernisen is dying. She was diagnosed at age 11 with a rare degenerative brain disorder that has no known cure or way to slow it down: juvenile-onset Huntington’s disease. “There’s lots of things that she used to be able to do just fine,” said her mom, Londen Tabor, who lives with her …

Dying Patients With Rare Diseases Struggle to Get Experimental Therapies Read More »

FDA update warns of potential rare nerve side effect for J&J Covid vaccine

The FDA is cautioning that the Johnson & Johnson Covid-19 vaccine may cause a rare immune response affecting the nerves. A causal link has not been established and the side effect, when observed, is rare, but the vaccine information has been updated to reflect the potential risk.

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

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 »

Bayer’s finerenone gets FDA nod for diabetic kidney disease

Bayer’s big investment in time and money on the development of finerenone for chronic kidney disease (CKD) in diabetics has paid off with an FDA approval, although it could face stiff competition in the market.  The oral, non-steroidal mineralocorticoid receptor (MR) antagonist has been approved as Kerendia in the US – its first world market …

Bayer’s finerenone gets FDA nod for diabetic kidney disease Read More »

Why We May Never Know Whether the $56,000-a-Year Alzheimer’s Drug Actually Works

The Food and Drug Administration’s approval in June of a drug purporting to slow the progression of Alzheimer’s disease was widely celebrated, but it also touched off alarms. There were worries in the scientific community about the drug’s mixed results in studies — the FDA’s own expert advisory panel was nearly unanimous in opposing its …

Why We May Never Know Whether the $56,000-a-Year Alzheimer’s Drug Actually Works Read More »

Aduhelm approval controversy dials up as FDA seeks probe

In another twist to the Aduhelm approval tale, FDA’s Janet Woodcock has called for an independent investigation into the relationship between agency staffers and Biogen executives. The acting FDA commissioner has sent a letter to the Department of Health and Human Services (HHS) inspector general Christi Grimm on Friday, asking for a review of the …

Aduhelm approval controversy dials up as FDA seeks probe Read More »

Bayer kidney disease drug gets FDA nod, pitting it against AstraZeneca, J&J meds

The FDA has approved Bayer’s Kerendia to treat chronic kidney disease in patients with type 2 diabetes. The drug is the first in its class, giving the pharma giant an alternative way to compete against medicines already marketed by AstraZeneca and Bayer.

Sigilon hemophilia A cell therapy trial placed on hold after patient complication

A hemophilia A patient in a Sigilon Therapeutics clinical trial developed a well-known complication to treatments for the blood disorder. The potential problem for Sigilon is that its cell therapy is designed to avoid triggering such immune responses.

FDA whittles back Aduhelm approval amid concern over broad label

One of the criticisms levelled at the FDA over its approval of Biogen and Eisai’s Alzheimer’s disease drug Aduhelm was its decision to clear use of the drug in a broader group of patients than was included in clinical trials. Now, it has backtracked on that decision. The updated label for Aduhelm (aducanumab) says it …

FDA whittles back Aduhelm approval amid concern over broad label Read More »

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

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 »

AZ, Amgen get speedy FDA review for asthma drug tezepelumab

The FDA has started a priority review of AstraZeneca and Amgen’s severe asthma antibody tezepelumab, setting a date for a decision in the first quarter of 2022. If approved, TSLP inhibitor tezepelumab could reach the market with a broader label than currently-used asthma antibodies, which are approved to treat patient with severe asthma characterised by …

AZ, Amgen get speedy FDA review for asthma drug tezepelumab Read More »

FDA rejects Provention’s type 1 diabetes drug teplizumab

The FDA indicated something was not well with Provention Bio’s filing for type 1 diabetes therapy teplizumab in April when it delayed its review. Now, the agency has rejected the application entirely, sending the company’s shares into a steep slide. The complete response letter (CRL) for teplizumab as a treatment to delay clinical type 1 …

FDA rejects Provention’s type 1 diabetes drug teplizumab Read More »

Sorrento’s Resiniferatoxin Receives the US FDA’s Clearance to Initiate P-II Trial for the Treatment of Knee Pain in Patients with Osteoarthritis

Shots: The P-II trial will assess the efficacy & safety of RTX across several dose groups vs PBO in patients with a mod. to sev. OAK pain. The study follows the P-Ib trial results of RTX The P-Ib data demonstrated the significant efficacy supporting RTX for long term refractory OA pain, pain relief observed in …

Sorrento’s Resiniferatoxin Receives the US FDA’s Clearance to Initiate P-II Trial for the Treatment of Knee Pain in Patients with Osteoarthritis Read More »

Provention Bio drug for delaying type 1 diabetes hit with FDA delay of its own

Provention Bio needs to show the FDA that the drug substance produced from its contract manufacturer is comparable to the Eli Lilly-made drug substance that was evaluated in clinical trials. A larger study testing the prospective type 1 diabetes therapy teplizumab is ongoing and could provide the information the agency needs.

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

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 »

Novartis tries to rescue stalled inclisiran filing with FDA

Novartis has tried to get its marketing application for high cholesterol therapy inclisiran in the US back on track, after the FDA rejected it last year, by changing the factory that makes the drug. The US regulator issued Novartis with a complete response letter (CRL) for inclisiran in December, citing “unresolved facility inspection-related conditions” at …

Novartis tries to rescue stalled inclisiran filing with FDA Read More »

Paying Billions for Controversial Alzheimer’s Drug? How About Funding This Instead?

If you could invest $56 billion each year in improving health care for older adults, how would you spend it? On a hugely expensive medication with questionable efficacy — or something else? This isn’t an abstract question. Aduhelm, a new Alzheimer’s drug approved by the Food and Drug Administration last month, could be prescribed to …

Paying Billions for Controversial Alzheimer’s Drug? How About Funding This Instead? Read More »

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

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 »

Merck pulls Keytruda’s FDA nod for third-line stomach cancer

Merck & Co will voluntarily withdraw a US marketing approval for cancer immunotherapy blockbuster Keytruda in gastric cancer, after studies intended to confirm its efficacy in this setting were a bust.  The decision comes after FDA advisors voted six to two in April that Keytruda (pembrolizumab) should not stay on the market as a treatment …

Merck pulls Keytruda’s FDA nod for third-line stomach cancer Read More »

The US FDA Approves FoundationOne CDx for Alunbrig (brigatinib) in Patients with ALK+ NSCLC

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 »

KHN’s ‘What the Health?’: Un-Trumping the ACA

Can’t see the audio player? Click here to listen on SoundCloud. You can also listen on Spotify, Apple Podcasts, Stitcher, Pocket Casts or wherever you listen to podcasts. The Biden administration this week proposed a series of changes aimed at boosting insurance enrollment under the Affordable Care Act, undoing changes made by the Trump administration …

KHN’s ‘What the Health?’: Un-Trumping the ACA Read More »

Jazz’s Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn) Receives the US FDA’s Approval for Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma

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 »

FDA rejection of rare disease drug leads Orphazyme to cut two thirds of staff

Orphazyme’s corporate restructuring comes less than two weeks after the FDA rejected arimoclomal, a drug developed to treat the rare Niemann-Pick disease type C. The company said it is now focused on securing regulatory approval in Europe and finding a path forward for the small molecule with the FDA.

Journalists Follow Up on Unused Vaccines and For-Profit Medical Schools

KHN correspondent Rachana Pradhan discussed the politics of unused Johnson & Johnson covid vaccines and the FDA’s potential announcement that their shelf life can be extended on Newsy on June 11. Click here to watch Pradhan on Newsy Read Pradhan’s “Unused Johnson & Johnson Covid Doses Are Piling Up as FDA Waits to See if …

Journalists Follow Up on Unused Vaccines and For-Profit Medical Schools Read More »

KHN’s ‘What the Health?’: The ACA Lives

Can’t see the audio player? Click here to listen on SoundCloud. You can also listen on Spotify, Apple Podcasts, Stitcher, Pocket Casts or wherever you listen to podcasts. For the third time in nine years, the Affordable Care Act has survived a constitutional challenge at the Supreme Court. In a 7-2 decision, the court ruled …

KHN’s ‘What the Health?’: The ACA Lives Read More »

Blueprint Medicines’ Ayvakit (avapritinib) Receives FDA’s Approval to Treat Patients with Advanced Systemic Mastocytosis

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 »

Not All Experts Are Ready to Vaccinate Kids Against Covid

Lucien Wiggins, 12, arrived at Tufts Children’s Hospital by ambulance June 7 with chest pains, dizziness and high levels of a protein in his blood that indicated inflammation of his heart. The symptoms had begun a day earlier, the morning after his second vaccination with the Pfizer-BioNTech mRNA shot. For Dr. Sara Ross, chief of …

Not All Experts Are Ready to Vaccinate Kids Against Covid 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

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 »

Consumer group seeks resignation of top FDA staffers over Aduhelm okay

With the first patient set to receive Biogen’s new Alzheimer’s drug Aduhelm in Rhode Island today, US consumer advocacy organisation Public Citizen is deeply unhappy about the FDA’s approval of the drug – and it wants the scalps of those responsible. In a letter to Secretary of Health and Human Services Xavier Becerra, the director …

Consumer group seeks resignation of top FDA staffers over Aduhelm okay Read More »

Gilead and Jounce’s JTX-1811 Receives US FDA’s IND Clearance for Cancer Immunotherapies

Shots: Jounce to receive $85M upfront and $35M in its common stock under the terms of Sep 2020 agreement. Additionally, it is eligible to receive upto $660M in future clinical, regulatory and commercial milestone payments along with royalties based upon the w/w sales after receiving a $25M milestone Jounce led the development of JTX-1811 through …

Gilead and Jounce’s JTX-1811 Receives US FDA’s IND Clearance for Cancer Immunotherapies Read More »

BeiGene’s Tislelizumab in Combination with Asieris’ APL-1202 Receive the US FDA’s IND Approval for MIBC

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 »

Novavax unveils phase 3 COVID jab data, but has it missed the boat in US?

The top-line phase 3 data for Novavax’ COVID-19 vaccine look good, with a 90% overall efficacy rate, but a dip in its share price indicates investors fear it could be too late for the programme. Novavax’ price spiked 9% after the data were announced today, but fell back just as quickly, despite the company’s assertions …

Novavax unveils phase 3 COVID jab data, but has it missed the boat in US? Read More »

J&J must ditch 60m COVID jabs made at US plant, but 10m are okay, says FDA

The failings at a Baltimore, US factory making Johnson & Johnson’s COVID-19 vaccine mean that millions of doses of the shot will have to be jettisoned, according to an FDA update. The problems at the plant run by contract manufacturer Emergent BioSolutions first emerged a few weeks ago, after an FDA inspection uncovered a string …

J&J must ditch 60m COVID jabs made at US plant, but 10m are okay, says FDA Read More »

Journalists Discuss New Alzheimer’s Drug, Women’s Alcohol Use, the Hip-Hop and Opioids Link

KHN chief Washington correspondent Julie Rovner discussed the FDA’s approval of a new drug for Alzheimer’s disease on WAMU’s “1A” on Wednesday. Click here to hear Rovner on WAMU KHN correspondent Aneri Pattani discussed the increase in alcohol use and misuse by young women on NPR’s “All Things Considered” on Wednesday. Click here to hear …

Journalists Discuss New Alzheimer’s Drug, Women’s Alcohol Use, the Hip-Hop and Opioids Link Read More »

And then there were three: another FDA adcomm member quits over Aduhelm

The fallout over the FDA’s decision to approved Biogen’s Alzheimer’s therapy Aduhelm continues to be felt, with a third member of its advisory committee resigning in protest.  The latest to depart the FDA’s Peripheral and Central Nervous System Drugs panel is Aaron Kesselheim of Harvard Medical School, joining fellow committee members Joel Perlmutter and David …

And then there were three: another FDA adcomm member quits over Aduhelm Read More »

Ocugen gets a black eye as FDA denies emergency OK for COVID jab

Ocugen is the first company to be affected by the FDA’s recent decision to raise the bar on emergency use authorisations for COVID-19 medicines, and will have to file for full approval of its vaccine candidate. That will hold back the biotech’s Covaxin shot by several months, and the delay put pressure on Ocugen’s share …

Ocugen gets a black eye as FDA denies emergency OK for COVID jab Read More »

Gilead’s Epclusa (Sofosbuvir/Velpatasvir) Receives the US FDA’s Approval for Children with Chronic Hepatitis C Infection

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 »

KHN’s ‘What the Health?’: Our 200th Episode!

Can’t see the audio player? Click here to listen on SoundCloud. You can also listen on Spotify, Apple Podcasts, Stitcher, Pocket Casts or wherever you listen to podcasts. The Food and Drug Administration found itself in the hot seat this week when it approved a controversial new drug to treat Alzheimer’s disease with scant evidence …

KHN’s ‘What the Health?’: Our 200th Episode! 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

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

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 wins FDA nod, getting edge in pneumococcus vax race—for now

The FDA has approved Prevnar 20, a Pfizer vaccine that protects against 20 pneumococcal strains—seven more than the company’s blockbuster Prevnar 13. But others are on Pfizer’s heels with vaccine candidates that aim to match or even beat the Pfizer pneumococcal vaccines.

Aduhelm approval could signal new era for CNS drugs; analysts

The unexpected approval and lofty pricing of Biogen’s Alzheimer’s therapy Aduhelm is clearly the biopharma event of the year so far, but what are the broader implications for the industry as a whole? Analysts at RBC Capital Markets say that the decision points to an unprecedented shift in position for the FDA when it comes …

Aduhelm approval could signal new era for CNS drugs; analysts Read More »

FDA clears Pfizer’s Prevnar 20 for adults, as Merck rival nears decision

Pfizer has the FDA approval it was seeking for Prevnar 20, the latest update to its blockbuster pneumococcal vaccination franchise, keeping it one step ahead of a competing shot from Merck & Co. The US regulator has cleared the pneumococcal conjugate vaccine – which protects against 20 common strains of Streptococcus pneumoniae – for adults …

FDA clears Pfizer’s Prevnar 20 for adults, as Merck rival nears decision 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

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 »

FDA slammed over Aduhelm OK as Biogen takes heat for $56k price tag

The FDA “has failed in its responsibility to protect patients and families from unproven treatments with known harms” in approving Biogen’s Alzheimer’s disease drug Aduhelm. That was the damning conclusion of the influential Institute for Clinical and Economic Review (ICER) in the US to yesterday’s approval of Aduhelm (aducanumab), the first new treatment for Alzheimer’s …

FDA slammed over Aduhelm OK as Biogen takes heat for $56k price tag Read More »

Biogen and Eisai’s Aduhelm (aducanumab-avwa) Receive the US FDA’s Accelerated Approval for Alzheimer Disease

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 »

Jubilation for Biogen, Eisai as FDA says ‘yes’ to aducanumab

Biogen and Eisai have defied the naysayers and secured an historic FDA approval for their amyloid-targeting antibody aducanumab as the first-ever disease-modifying treatment for Alzheimer’s disease. The accelerated approval from the US regulator has been given even though its own advisory committee comprehensively voted down the main study filed in support of the drug last …

Jubilation for Biogen, Eisai as FDA says ‘yes’ to aducanumab Read More »

Novo Nordisk’s Wegovy (semaglutide, 2.4mg) Receives the US FDA’s Approval for Weight Management in Adults with Obesity

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 »

FDA authorizes Regeneron’s Covid drug at lower dose, and in injectable formulation

Emergency authorization of Regeneron Pharmaceuticals’ Covid-19 antibody drug now includes an injectable version, which is a more convenient option for patients. The drug developer plans to seek full FDA approval of its antibody cocktail later this summer.

Dermavant Submits NDA to FDA for Tapinarof Cream to Treat Plaque Psoriasis

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 »

FDA Weighs Approval of a Lucrative Alzheimer’s Drug but Benefits Are Iffy

The Food and Drug Administration’s decision next week whether to approve the first treatment for Alzheimer’s disease highlights a deep division over the drug’s benefits as well as criticism about the integrity of the FDA approval process. This story also ran on The Daily Beast. It can be republished for free. The agency said it …

FDA Weighs Approval of a Lucrative Alzheimer’s Drug but Benefits Are Iffy Read More »

FDA grants swift approval to Cognoa’s digital device for autism diagnosis

The FDA has approved a digital device developed by Cognoa that can be used to diagnose autism spectrum disorder (ASD), just a few weeks after it was filed for approval. Cognoa says the artificial intelligence (AI) based device is the first to be authorised by the US regulator that can help doctors diagnose autism in …

FDA grants swift approval to Cognoa’s digital device for autism diagnosis Read More »

Cognoa Awarded FDA Authorization For AI-Based Autism Diagnosis Aid

What You Should Know: – Cognoa, a pediatric behavioral health company developing diagnostic and therapeutic solutions for children living with behavioral health conditions, has been granted FDA authorization to market the company’s first-of-its-kind, AI-based autism diagnosis aid – Canvas Dx. – Canvas Dx is the first diagnosis aid designed to help physicians to diagnose autism spectrum disorder (ASD) in the primary care setting, as well as the first diagnosis aid for ASD authorized for commercialization by …

Cognoa Awarded FDA Authorization For AI-Based Autism Diagnosis Aid Read More »

Janssen’s Teclistamab Receives the US FDA’s Breakthrough Therapy Designation for Relapsed or Refractory Multiple Myeloma

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

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 »

Bouncing back from FDA rejection, Alkermes psychiatric drug wins approval

Alkermes antipsychotic drug Lybalvi now has FDA approval. Though the field of neuropsychiatric drugs is crowded with generic medications, Alkermes believes Lybalvi, designed to mitigate the weight gain side effect common with mood disorder drugs, could have an advantage.

FDA faces big questions as it takes a closer look at AI regulations

An action plan shared by the FDA earlier this year showed how the regulator is thinking about future regulations for AI and machine learning in healthcare. But the agency still has several big questions to address as it prepares to roll out new guidance.

Amgen nabs speedy approval for first-ever KRAS drug sotorasib

After decades of mostly unsuccessful R&D, Amgen has claimed the first ever regulatory approval for a drug that inhibits KRAS – a cancer target that for a long time was all-but written off as “undruggable”. The FDA’s conditional approval for Lumakras (sotorasib; formerly AMG-510) as a second-line treatment for KRAS G12c-mutated non-small cell lung cancer …

Amgen nabs speedy approval for first-ever KRAS drug sotorasib Read More »

Ipsen Reports the US FDA’s Acceptance of NDA for Palovarotene to Treat Fibrodysplasia Ossificans Progressiva

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)

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

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 »

Amgen’s Lumakras (sotorasib) Receives the US FDA’s Approval as the First KRAS Blocking Cancer Therapy

Shots: The approval is based on the CodeBreaK 100 trial evaluating the efficacy & tolerability of Lumakras (960mg, qd) in 124 patients with KRAS G12C mutation-positive NSCLC whose disease had progressed after prior treatment with immunotherapy or CT Results: ORR (36%); DCR (81%); mDoR (10 mos.); durable response with a positive benefit-risk profile, 9% showed …

Amgen’s Lumakras (sotorasib) Receives the US FDA’s Approval as the First KRAS Blocking Cancer Therapy Read More »

Amgen lands first FDA approval for drug targeting elusive mutation in lung cancer

FDA approval of Amgen drug sotorasib (Lumakras) makes the small molecule the first compound authorized by the regulator for treating cancers characterized by a KRAS G12C mutation. Mutated KRAS proteins had long been regarded “undruggable” targets.

The US FDA’s EMDAC Supports Provention Bio’s Teplizumab in Delaying Diabetes

Shots: EMDAC has voted 10-7, confirming that the benefits of teplizumab outweigh the risks supporting the approval to delay T1D Mellitus The recommendation is based on a pivotal TN-10 study in which a single 14-day course of teplizumab delayed insulin-dependent, clinical-stage disease by a median of at least 2yrs. in pre symptomatic patients with stage …

The US FDA’s EMDAC Supports Provention Bio’s Teplizumab in Delaying Diabetes Read More »

BMS’ Zeposia (ozanimod) Receives the US FDA’s Approval for Ulcerative Colitis

Shots: The approval is based on a P-III True North trial assess Zeposia (0.92 mg) vs PBO in an adult with mod. to sev. active UC The trial met its 1EPs & 2EPs i.e Zeposia as an induction & maintenance therapy demonstrates clinical remission (18% vs 6% & 37% vs 19%), clinical response (48% vs …

BMS’ Zeposia (ozanimod) Receives the US FDA’s Approval for Ulcerative Colitis Read More »

FDA authorizes Vir & GSK Covid-19 drug, introducing third antibody option

FDA authorization of the Covid-19 antibody drug from Vir Biotechnology and GlaxoSmithKline adds a third antibody drug for treating early cases of the disease that are at risk of progressing to hospitalization. The companies say they plan to launch the drug, named sotrovimab, “in coming weeks.”

MedCity Pivot Podcast: The stakes are high for getting AI right in healthcare, says Duke Forge director

It’s important for clinicians to consider AI recommendations with a critical eye, the same as they would for any other test, said Dr. Erich Huang, Director of Duke Forge. In our latest podcast episode, he spells out the stakes for implementing algorithms in healthcare, which can potentially affect thousands of patients.

GSK, Sanofi eye Q4 approval as COVID jab starts phase 3

GlaxoSmithKline’s COVID-19 drive has advanced on two fronts, with the start of phase 3 trials of its Sanofi-partnered vaccine candidate as well as an FDA emergency approval for antibody sotrovimab, developed with Vir Biotech. GSK and Sanofi think they could be on track for approval of the recombinant protein-based vaccine – delivered with GSK’s immune-response …

GSK, Sanofi eye Q4 approval as COVID jab starts phase 3 Read More »

Pfizer and Myovant’s Myfembree Receive the US FDA’s Approval for Heavy Menstrual Bleeding Associated with Uterine Fibroids

Shots: The approval is based on efficacy and safety data from the P-III LIBERTY 1 & 2 studies assessing Myfembree (relugolix 40 mg, estradiol 1 mg & norethindrone acetate 0.5 mg) vs PBO for heavy menstrual bleeding associated with uterine fibroids in premenopausal women with a treatment duration for up to 2yrs. The P-III studies …

Pfizer and Myovant’s Myfembree Receive the US FDA’s Approval for Heavy Menstrual Bleeding Associated with Uterine Fibroids Read More »

GSK and Vir Receive the US FDA’s EUA for Sotrovimab (VIR-7831) to Treat COVID-19 in Adults and Pediatric Patients

Shots: The US FDA has granted EUA for sotrovimab for mild to mod. COVID-19 patients aged ≥12yrs. who are at high risk for progression to severe COVID-19 including hospitalization for >24hrs. or death The EUA is based on an interim analysis from the P-III COMET-ICE trial that demonstrated an 85% reduction in hospitalization or death. …

GSK and Vir Receive the US FDA’s EUA for Sotrovimab (VIR-7831) to Treat COVID-19 in Adults and Pediatric Patients Read More »

FDA places clinical hold on Larimar’s rare disease drug after deaths in monkey study

The FDA placed a clinical hold on a Larimar Therapeutics program for Friedreich’s ataxia after monkeys died in tests of the fusion protein. In addition to halting further tests of the drug, the clinical hold also ends the biotech’s plan to raise $95 million that would have funded clinical research.

TG Therapeutics Reports the US FDA’s Acceptance of BLA for Ublituximab + Ukoniq (umbralisib) to Treat Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma

Shots: The BLA submission is based on P-III UNITY-CLL trial evaluating ublituximab + Ukoniq (U2) vs obinutuzumab + chlorambucil in a ratio (1:1) in 420 patients with treatment-naive and r/r CLL. The 1EP of the study was PFS The trial is being conducted under a SPA agreement with the US FDA. The FDA also notified …

TG Therapeutics Reports the US FDA’s Acceptance of BLA for Ublituximab + Ukoniq (umbralisib) to Treat Chronic Lymphocytic Leukemia and Small Lymphocytic Lymphoma Read More »

Moderna’s Covid vaccine shows efficacy in younger teens; regulatory filing are next

Moderna released preliminary clinical data showing its Covid-19 vaccine produced efficacy in adolescents that was comparable to what was seen in adults. The company plans to submit applications to regulators in early June seeking expansion of emergency authorization of the vaccine to include this younger age group.

Boehringer Ingelheim’s BI 425809 Receives the US FDA’s Breakthrough Therapy Designation for Cognitive Impairment Associated with Schizophrenia

Shots: The BTD is based on P-II 1346.9 clinical trial, which demonstrated that BI 425809 improved cognition in adult patients with schizophrenia Moreover, the company has initiated the P-III CONNEX program assessing the safety and efficacy of BI 425809 vs PBO for improving cognition in adults with schizophrenia over a 26wks. treatment period Additionally, Boehringer …

Boehringer Ingelheim’s BI 425809 Receives the US FDA’s Breakthrough Therapy Designation for Cognitive Impairment Associated with Schizophrenia Read More »

FDA approval of J&J drug is first for lung cancer with particular genetic mutation

Johnson & Johnson subsidiary Janssen was awarded approval for Rybrevant, the latest in a string of decisions for targeted therapies for non-small cell lung cancer. Rybrevant is the first approved targeted therapy addressing a subset of patients whose disease is characterized by a particular mutation to cancer protein EGFR.

Janssen’s Rybrevant (amivantamab-vmjw) Receives the US FDA’s Approval as the First Targeted Treatment for Patients with NSCLC with EGFR Exon 20 Insertion Mutations

Shots: The US FDA has granted accelerated approval which is based on P-I CHRYSALIS study assessing Rybrevant as a monothx. in patients metastatic non-small cell lung cancer with epidermal growth factor receptor exon 20 insertion mutations whose disease has progressed on or after platinum-based CT The FDA has also approved Guardant Health’s Guardant360 CDx liquid …

Janssen’s Rybrevant (amivantamab-vmjw) Receives the US FDA’s Approval as the First Targeted Treatment for Patients with NSCLC with EGFR Exon 20 Insertion Mutations Read More »

BMS Receive the CHMP’s Positive Opinion Recommending Approval of Opdivo (nivolumab) + Yervoy (ipilimumab) for dMMR or MSI-H mCRC After Prior Chemotherapy

Shots: The opinion is based on P-II CheckMate -142 study assessing Opdivo + Yervoy in patients with mismatch repair deficient or microsatellite instability–high metastatic colorectal cancer whose disease had progressed during or after prior treatment with CT If approved, Opdivo in combination with Yervoy will be the first dual immunotherapy approved in the EU in …

BMS Receive the CHMP’s Positive Opinion Recommending Approval of Opdivo (nivolumab) + Yervoy (ipilimumab) for dMMR or MSI-H mCRC After Prior Chemotherapy Read More »

Should you adjust for covariates when analyzing data from randomized controlled trials?

FDA draft guidance published this month says you should. In most cases, adjusting for covariates is not necessary. Randomization generally insurers that covariates are balanced across clinical trial arms. Randomization, however, may not always result in perfectly balanced trial arms. In these cases, the FDA notes that covariate adjustment is perfectly acceptable. There are some …

Should you adjust for covariates when analyzing data from randomized controlled trials? Read More »

Lycopene Supplements Put to the Test

High doses of lycopene—the red pigment in tomatoes—were put to the test to see if it could prevent precancerous prostate lesions from turning into full-blown cancer. Back in 1980s, the Adventist Health Study found “strong protective relationships” against prostate cancer with increasing consumption of legumes, citrus, dried fruit, nuts, and tomatoes. In the 1990s, a …

Lycopene Supplements Put to the Test Read More »

BioXcel Reports the US FDA’s Acceptance of BXCL501’s NDA for the Acute Treatment of Agitation Associated with Schizophrenia and Bipolar Disorders I and II

Shots: The NDA submission is based on P-III studies (SERENITY I & II) assessing BXCL501 (120 & 180mcg doses) vs PBO for the acute treatment of agitation associated with schizophrenia and bipolar disorders I and II, respectively Results: BXCL501 met its 1EPs & 2EPs in both the studies, demonstrating rapid, and durable improvements from baseline …

BioXcel Reports the US FDA’s Acceptance of BXCL501’s NDA for the Acute Treatment of Agitation Associated with Schizophrenia and Bipolar Disorders I and II Read More »

FDA fires broadside at CytoDyn over COVID-19 antibody data

In a rare move, the FDA has publicly accused US biotech CytoDyn of misrepresenting clinical trial results for leronlimab, an antibody being developed for COVID-19.  It’s not uncommon for drug developers put a positive spin on lacklustre clinical data, but CytoDyn’s attempts seems to have irritated the US regulator, specifically relating to its use of …

FDA fires broadside at CytoDyn over COVID-19 antibody data Read More »

Glaukos Expands its Partnership with Santen for Preserflo MicroShunt

Shots: The companies enter into a license agreement for the Preserflo MicroShunt superseding the previous collaboration between the two parties Glaukos get exclusive commercialization rights for the MicroShunt in the US, Australia, New Zealand, Canada, Brazil, Mexico and the remainder of Latin America and gain full control over all development activities for the MicroShunt in …

Glaukos Expands its Partnership with Santen for Preserflo MicroShunt Read More »

BMS Signs an Exclusive Global License with Agenus for its Anti-TIGIT Bispecific Antibody Program

Shots: Agenus to receive a $200M as up front & ~$1.36B as development, regulatory and commercial milestones plus royalties on net sales of the therapies. Agenus will retain options to conduct clinical studies and combination studies with other pipeline assets and to co-promote AGEN1777 in the US BMS will be solely responsible for the development …

BMS Signs an Exclusive Global License with Agenus for its Anti-TIGIT Bispecific Antibody Program Read More »

How strengthening the biosimilar marketplace benefits patients

Biologics are medicines that are made from living organisms through highly complex manufacturing processes and include a wide variety of medicines such as therapeutic proteins, monoclonal antibodies and vaccines. They are used to prevent or treat a variety of diseases including cancer, chronic kidney disease, diabetes, cystic fibrosis and autoimmune disorders.  

Medicago and GSK Report Results of Plant-Derived Vaccine in a P-II Study for COVID-19

Shots: The P-II study involves assessing the safety & immunogenicity of the CoVLP vs PBO in 306 subjects aged ≥18yrs. with two age groups (18-64yrs & over 65yrs.) across the US & Canada Results: vaccine induced humoral immune response of similar strength in both age cohorts after two doses, both age cohorts responded with NAb …

Medicago and GSK Report Results of Plant-Derived Vaccine in a P-II Study for COVID-19 Read More »

Roche Receives the US FDA’s EUA for cobas SARS-CoV-2 Test to Suspect COVID-19

Shots: Roche’s cobas SARS-CoV-2 test to be use on high-throughput cobas 6800/8800 systems has received the US FDA’s EUA for testing individuals without symptoms or reasons to control the spread of COVID-19 The authorization expands SARS-CoV-2 testing to include people without symptoms and applies to pooled samples containing up to and including 6 individual samples …

Roche Receives the US FDA’s EUA for cobas SARS-CoV-2 Test to Suspect COVID-19 Read More »

Eli Lilly and Innovent Report the US FDA’s Acceptance of Sintilimab + Chemotherapy for 1L Treatment for Non-Squamous NSCLC

Shots: The submission is based on a P-III ORIENT-11 study assessing sintilimab (200mg) + pemetrexed and platinum CT vs PBO + CT as a 1L treatment for 397 patients in a ratio (2:1) with advanced or metastatic nonsq. NSCLC, with no sensitizing EGFR mutations or ALK rearrangements The anticipated PDUFA date for this application is …

Eli Lilly and Innovent Report the US FDA’s Acceptance of Sintilimab + Chemotherapy for 1L Treatment for Non-Squamous NSCLC Read More »

CMS’ rule granting speedy coverage to FDA-designated ‘breakthrough’ devices delayed again

CMS delayed the start date of its rule that would provide expedited Medicare coverage for products the FDA deems “breakthrough devices” from May 15 to Dec. 15. CMS wants more time to examine concerns expressed about the rule, including that devices may gain coverage despite limited evidence of their use among seniors.

With FDA approval, Apellis can challenge AstraZeneca in rare blood disorder

The FDA approved Apellis Pharmaceuticals drug pegcetacoplan (Empaveli) as a treatment for a rare blood disorder that is currently treated with medicines sold by Alexion Pharmaceuticals, a biotech being acquired by AstraZeneca. But Apellis’s drug beat Alexion drug Soliris in a head-to-head test, and the smaller company aims to steal market share from its larger …

With FDA approval, Apellis can challenge AstraZeneca in rare blood disorder Read More »

Apellis’ Empaveli (pegcetacoplan) Receives the US FDA’s Approval for Paroxysmal Nocturnal Hemoglobinuria

Shots: The approval is based on P-III head-to-head PEGASUS study assessing Empavel vs Soliris (eculizumab) in 80 adults with PNH. Participants should be Soliris stable for at least 3mos. with a Hgb level of <10.5 g/dL at the screening visit Results: The study met its 1EPs demonstrating superiority to Soliris for the change from baseline …

Apellis’ Empaveli (pegcetacoplan) Receives the US FDA’s Approval for Paroxysmal Nocturnal Hemoglobinuria Read More »

Talking about Cybersecurity Vulnerabilities in Medical Devices Shouldn’t be Taboo

Nastassia Tamari, Director of Information Security Operations for BD According to the National Vulnerability Database, 18,353 vulnerabilities were reported in 2020. That’s nearly three times the volume of vulnerabilities reported five years ago, and higher than any year in the previous two decades. Given the rise in connected devices, this increase is not entirely unexpected. …

Talking about Cybersecurity Vulnerabilities in Medical Devices Shouldn’t be Taboo Read More »

Heron Therapeutics’ ZYNRELEF (HTX-011) Receives the US FDA’s Approval for the Management of Postoperative Pain

Shots: The US FDA approved Zynrelef (bupivacaine and meloxicam) extended-release solution for postsurgical analgesia in patients with soft tissue or periarticular instillation for 72 hrs after a bunionectomy, open inguinal herniorrhaphy and total knee arthroplasty The approval is based on data from the P-III studies assessing Zynrelef vs bupivacaine solution in 1000 patients which demonstrated …

Heron Therapeutics’ ZYNRELEF (HTX-011) Receives the US FDA’s Approval for the Management of Postoperative Pain Read More »

KHN’s ‘What the Health?’: Drug Price Effort Hits a Snag

Can’t see the audio player? Click here to listen on SoundCloud. The high cost of prescription drugs is a top health issue for the public and politicians, but concerns raised by a group of moderate Democrats threaten to derail a bill being pushed by House Democratic leaders. Meanwhile, the Food and Drug Administration has authorized the …

KHN’s ‘What the Health?’: Drug Price Effort Hits a Snag Read More »

Heron Therapeutics’ combo drug for post-surgical pain wins FDA nod on third try

Heron Therapeutics is positioning its drug, Zynrelef, as a way to help patients avoid addictive opioid painkillers following surgery. The extended-release formulation offers analgesic effects over 72 hours—when postsurgical pain is most severe and more likely to require pain management medication.

FDA Approves First Ortho-K Contact Lens to Manage Myopia

What You Should Know: – Today, Johnson & Johnson Vision announced that the U.S. Food and Drug Administration (FDA) has approved ACUVUE® Abiliti™ Overnight Therapeutic Lenses, the first and only orthokeratology (ortho-k) contact lens for the management of myopia.   – Myopia, or ‘nearsightedness,’ is a chronic and progressive disease. Research shows that myopia will affect half of the world’s population by 2050, making it the biggest eye health …

FDA Approves First Ortho-K Contact Lens to Manage Myopia Read More »