Us FDA

Medical Writing — An Outsourcing Competency

The history of medical writing began with the rise of the modern clinical trial. A medical writer can be your most pivotal ally in a clinical trial. While clinic directors, data managers, and investigators in the pharmaceutical industry are required for clinical trials, medical writers are the glue that holds clinical trials together. The increase …

Medical Writing — An Outsourcing Competency Read More »

Fulcrum Therapeutics’ Losmapimod Receives the US FDA’s Fast Track Designation to Treat Facioscapulohumeral Muscular Dystrophy

Shots: The US FDA has granted FTD to losmapimod for the treatment of FSHD while the therapy has previously received ODD for FSHD The company will present full data from the P-IIb ReDUX4 trial assessing losmapimod vs PBO in patients with FSHD at FSHD on June 24-25, 2021 that include 1EP results i.e., reduction from …

Fulcrum Therapeutics’ Losmapimod Receives the US FDA’s Fast Track Designation to Treat Facioscapulohumeral Muscular Dystrophy Read More »

Merck and Eisai Receive the US FDA‘s Priority Review for Keytruda (pembrolizumab) + Lenvima (lenvatinib) Applications to Treat Advanced Renal Cell Carcinoma and Endometrial Carcinoma

Shots: The 1st sNDA and sBLA is based on results from the P-III CLEAR Study evaluating Lenvima + Keytruda or in combination with everolimus vs sunitinib in a ratio (1:1:1) in 1,069 patients with advanced RCC, demonstrated improved PFS, OS, and ORR The 2nd application is based on results from the P-III KEYNOTE-775/Study 309 trial …

Merck and Eisai Receive the US FDA‘s Priority Review for Keytruda (pembrolizumab) + Lenvima (lenvatinib) Applications to Treat Advanced Renal Cell Carcinoma and Endometrial Carcinoma Read More »

Roche Receives the US FDA’s Approval for First CDx to Identify Endometrial Cancer Patients Eligible for Immunotherapy

Shots: The US FDA has approved Ventana MMR RxDx panel to identify endometrial cancer patients who are eligible for treatment with GSK’s Jemperli (monothx.) that has received the US FDA’s approval on Apr 22, 2021 The CDx provides clinicians with a standardized testing option that uses a comprehensive panel of DNA mismatch repair (MMR) biomarkers …

Roche Receives the US FDA’s Approval for First CDx to Identify Endometrial Cancer Patients Eligible for Immunotherapy Read More »

AbbVie Submits Regulatory Applications to the US FDA and EMA for Skyrizi (risankizumab) to Treat Psoriatic Arthritis

Shots: The submission is based on two P-III studies KEEPsAKE-1 & -2 studies involves assessing Skyrizi (150mg) vs PBO in adult patients with active PsA who had an inadequate response or intolerant to biologic therapy and/or non-biologic DMARDs respectively The 1EP and 2EP of both studies showed ACR20 response @24wks., improvements in disease activity, skin …

AbbVie Submits Regulatory Applications to the US FDA and EMA for Skyrizi (risankizumab) to Treat Psoriatic Arthritis Read More »

Coherus Reports the US FDA’s Acceptance of BLA for CHS-1420 (biosimilar, adalimumab)

Shots: The US FDA has accepted the BLA under the 351(k) for CHS-1420 with an anticipated BsUFA date as Dec’2021 The company plans to launch the CHS-1420 in the US on or after Jul 01, 2023 as per the terms of an agreement with AbbVie Coherus collaborated with Junshi in Feb’2021 to in-license toripalimab in …

Coherus Reports the US FDA’s Acceptance of BLA for CHS-1420 (biosimilar, adalimumab) Read More »

Pfizer and OPKO Reports the US FDA’s Acceptance of BLA for Somatrogon to Treat Pediatric Patients with Growth Hormone Deficiency

Shots: The BLA submission is based on P-lll study assessing the safety and efficacy of Somatrogon (0.66 mg/kg, qw) vs Somatropin (0.034 mg/kg, qd) in 224 pediatric patients in the ratio of (1:1) with GHD The study met its 1EPs of non-inferiority as measured by annual height velocity @12mos.; least square mean (10.12 vs 9.78 …

Pfizer and OPKO Reports the US FDA’s Acceptance of BLA for Somatrogon to Treat Pediatric Patients with Growth Hormone Deficiency Read More »

The US FDA Grants EUA for Quanterix’s Simoa Semi-Quantitative SARS-CoV-2 IgG Antibody Test

Shots: The FDA has issued a EUA for Simoa Semi-Quantitative SARS-CoV-2 IgG Ab test and can be operated on Simoa HD-X Analyzer The test checks for Abs that are directed against the spike protein. The test delivers a numerical result demonstrating Ab concentration ranging from 0.21-250mg/mL. The clinical studies demonstrated that the test had a …

The US FDA Grants EUA for Quanterix’s Simoa Semi-Quantitative SARS-CoV-2 IgG Antibody Test Read More »

Chi-Med Initiates Rolling Submission of NDA to the US FDA of Surufatinib to Treat Advanced Neuroendocrine Tumors

Shots: Chi-Med initiated the filing of NDA to the US FDA for surufatinib to treat pancreatic & non-pancreatic NET & plans to complete the NDA submission in the H1’21 The NDA is based on two P-III studies that demonstrated a reduction in risk of progression or death by 67% & 51%, extending PFS of non-pancreatic …

Chi-Med Initiates Rolling Submission of NDA to the US FDA of Surufatinib to Treat Advanced Neuroendocrine Tumors Read More »

Ocular Therapeutix Reports sNDA Submission of Dextenza (dexamethasone ophthalmic insert) to the US FDA for Ocular Itching

Shots: The sNDA submission is based on P-II/P-III Clinical trial assessing Dextenza(dexamethasone ophthalmic insert, 0.4mg) vs PBO for the treatment of Ocular Itching associated with allergic conjunctivitis Result: At 1EPs for the pooled analysis of P-III clinical trial at Day 8; ocular itching favored DEXTENZA treated participants compared with PBO at all 3-time points: 3 …

Ocular Therapeutix Reports sNDA Submission of Dextenza (dexamethasone ophthalmic insert) to the US FDA for Ocular Itching Read More »

Janssen Initiates Rolling Submission of BLA to US FDA of Autoleucel (cilta-cel) to Treat Multiple Myeloma

Shots: Janssen Initiates Rolling Submission of BLA to US FDA is based on P-lb/ll CARTITUDE-1 Study involve evaluating the safety and efficacy of Autoleucel (cilta-cel) in adults for the treatment of relapsed and refractory Multiple Myeloma The FDA granted BTD for cilta-cel and agreed to BLA’s rolling review includes completed portions of the application to …

Janssen Initiates Rolling Submission of BLA to US FDA of Autoleucel (cilta-cel) to Treat Multiple Myeloma Read More »

Moderna’s mRNA-1273 Receives the US FDA’s EUA Against COVID-19

Shots: The US FDA has authorized EUA of mRNA-1273 vaccine against COVID-19 in individuals ≥18yrs. The delivery to the US govt. will begin imminently while Moderna will continue to gather additional data and plans to file BLA for full licensure in 2021 Allocation and distribution will be prioritized according to populations identified by the CDC’s …

Moderna’s mRNA-1273 Receives the US FDA’s EUA Against COVID-19 Read More »

Amgen’s Riabni (biosimilar, rituximab) Receives the US FDA’s Approval for Multiple Diseases

Shots: The approval is based on trial assessing Riabni (375 mg/m2, IV) vs Rituxan once weekly for 4wks. followed by dosing @12wks. & 20wks. in 256 patients in a ratio (1:1) with grade 1, 2, or 3a follicular B-cell NHL & low tumor burden The WAC of Riabni will be 23.7% lower than the Rituxan …

Amgen’s Riabni (biosimilar, rituximab) Receives the US FDA’s Approval for Multiple Diseases Read More »

Moderna’s mRNA-1273 Receives the US FDA’s Advisory Committee Vote Supporting EUA Against COVID-19

Shots: The VRBPAC voted 20-0, with one abstention that the benefits of the Moderna’s vaccine outweighed its risks for use in people aged ≥18yrs The recommendation is based on data analysis from the pivotal P-III clinical study that demonstrated 94.1% efficacy The US FDA’s committees provide non-binding recommendations. The FDA will take the VRBPAC’s recommendation …

Moderna’s mRNA-1273 Receives the US FDA’s Advisory Committee Vote Supporting EUA Against COVID-19 Read More »

Amgen Reports NDA Submission of Sotorasib to the US FDA for Advanced/Metastatic NSCLC With KRAS G12C Mutation

Shots: The NDA submission is based P-II CodeBreaK 100 study assessing Sotorasib in patients with KRAS G12C-mutated NSCLC prior treated with CT and immunotherapy The study provided durable anticancer activity with a positive benefit-risk profile The US FDA has reviewed the NDA under RTOR pilot program, which aims to explore efficient review process that ensures …

Amgen Reports NDA Submission of Sotorasib to the US FDA for Advanced/Metastatic NSCLC With KRAS G12C Mutation Read More »

Ellume’s COVID-19 Home Test Receives the US FDA’s EUA as First Over-the-Counter Diagnostic Test

Shots: The US FDA has granted EUA for Ellume COVID-19 home test for non-prescription home use in symptomatic and asymptomatic individuals aged ≥2yrs. The data submitted to the US FDA from an independently run, simulated home-setting clinical study of 198 subjects aged 2-82yrs, demonstrated 96% accuracy including 95% overall sensitivity & 97% specificity vs emergency …

Ellume’s COVID-19 Home Test Receives the US FDA’s EUA as First Over-the-Counter Diagnostic Test Read More »

Genentech’s Ocrevus Shorter 2-Hour Infusion Receives the US FDA’s Approval for Relapsing & Primary Progressive Multiple Sclerosis

Shots: The approval is based on ENSEMBLE PLUS study, which demonstrated similar frequency and severity of IRRs for 2hrs. Ocrevus infusion time vs conventional 3.5hrs in patients with RRMS. The initial dose is given as two 300mg infusions given 2wks. apart and a subsequent dose of single 600mg infusions were administered over a shorter, 2hrs. …

Genentech’s Ocrevus Shorter 2-Hour Infusion Receives the US FDA’s Approval for Relapsing & Primary Progressive Multiple Sclerosis Read More »

Pfizer and BioNTech’s BNT162b2 Receives the US FDA’s EUA to Prevent COVID-19

Shots: Pfizer & BioNTech received the US FDA’s EUA for BNT162b2 for patients aged ≥16yrs with COVID-19. The companies are gathering additional data & prepared to file a planned BLA with the US FDA anticipating full approval in 2021 The EUA is based on P-III pivotal study demonstrating 95% efficacy in participants without prior SARS-CoV-2 …

Pfizer and BioNTech’s BNT162b2 Receives the US FDA’s EUA to Prevent COVID-19 Read More »

Genentech and Novartis’s Xolair (omalizumab) Receives the FDA’s Approval for Adults with Nasal Polyps

Shots: The approval is based on P-III POLYP 1 & 2 trials assessing Xolair vs PBO in 138 & 127 adult patients with nasal polyps who had an inadequate response to nasal corticosteroids respectively Results: @24wks. improvement in NPS (-1.1 vs 0.1 & -0.9 vs -0.3); improvement in NCS (-0.9 vs -0.4 & -0.7 vs …

Genentech and Novartis’s Xolair (omalizumab) Receives the FDA’s Approval for Adults with Nasal Polyps Read More »

Moderna to Seek the FDA and EMA’s EUA for mRNA-1273 and Shows 100% Efficacy in P-III COVE-Study

Shots: The P-III COVE study demonstrated 94.1% efficacy against COVID-19 and 100% efficacy against severe COVID-19. The vaccine was well tolerated with no serious safety concerns identified to date Moderna plans to submit an EUA to the FDA & an application for CMA with the EMA and to progress with the rolling reviews, which have …

Moderna to Seek the FDA and EMA’s EUA for mRNA-1273 and Shows 100% Efficacy in P-III COVE-Study Read More »

CorticoMetrics’s THINQ Receives the US FDA’s 510(k) Clearance for MRI Brain Volumetric Reporting

Shots: The US FDA has granted 510(k) clearance for CorticoMetrics’ AI-based software, THINQ, for MRI brain volumetric reporting. The software can provide radiologists & neurologists with quantitative to improve assessment of neurological disorders The software can automatically segment T1-weighted MRI brain image, providing volumetric measurements & visualisations of 30 critical brain structures and compares the …

CorticoMetrics’s THINQ Receives the US FDA’s 510(k) Clearance for MRI Brain Volumetric Reporting Read More »

Alnylam’s Oxlumo (lumasiran) Receives the US FDA’s Approval for Treatment of Primary Hyperoxaluria Type 1 in Pediatric and Adult Patients

Shots: The approval is based on P-III ILLUMINATE-A & -B trials. The studies demonstrating reductions in urinary oxalate and encourage safety and tolerability in pediatric and adult patients The ILLUMINATE-A showed that Oxlumo met its 1EP i.e. change in 24hrs. (65% vs 12%) compared to PBO, the study also achieved significant results for all 6 …

Alnylam’s Oxlumo (lumasiran) Receives the US FDA’s Approval for Treatment of Primary Hyperoxaluria Type 1 in Pediatric and Adult Patients Read More »

Roche’s Xofluza (baloxavir marboxil) Receives the US FDA’s Approval for Post-Exposure Prevention of Influenza

Shots: The approval is based on P-III BLOCKSTONE study assessing a single dose of Xofluza vs PBO in household members who were living with someone with influenza confirmed by a rapid influenza diagnostic test Results: The proportion of household members aged ≥12yrs. who developed influenza (1% vs 13%), well-tolerated with no new safety signals Xofluza …

Roche’s Xofluza (baloxavir marboxil) Receives the US FDA’s Approval for Post-Exposure Prevention of Influenza Read More »

Eiger’s Zokinvy (lonafarnib) Receives the US FDA’s Approval for Treatment of Progeria and Processing-Deficient PL

Shots: The Progeria Research Foundation and Eiger reported the US FDA’s approval of Zokinvy (lonafarnib) for the treatment of HGPS or progeria and processing-deficient progeroid laminopathies Zokinvy reduced the incidence of mortality by 60% & increased the average survival time by 2.5 yrs. Additionally, the FDA has issued an RPD priority review voucher to Eiger …

Eiger’s Zokinvy (lonafarnib) Receives the US FDA’s Approval for Treatment of Progeria and Processing-Deficient PL Read More »

Alvotech Reports the US FDA and EMA’s Acceptance of AVT02 Proposed Biosimilar to Humira (adalimumab)

Shots: The US FDA has accepted the BLA of AVT02 for review and is expected to decide on the filing in Sept’2021 while the EMA has accepted for review an MAA for AVT02 with an EMA decision anticipated in the Q4’21 The filings were based on AVT02-GL-101 & AVT02-GL-301 studies demonstrating a high degree of …

Alvotech Reports the US FDA and EMA’s Acceptance of AVT02 Proposed Biosimilar to Humira (adalimumab) Read More »

Chiesi’s Bronchitol (mannitol) Inhalation Powder Receives the US FDA’s Approval for Cystic Fibrosis

Shots: In the three large-scale global clinical trials assessing Bronchitol in 761 patients, sustained improvement in FEV1 (Forced Expiratory Volume) with Bronchitol use vs. control group was observed Bronchitol is currently approved and marketed in Australia, Italy, Germany, Russia, and several other countries. Additionally, the company anticipates launching Bronchitol in the US in Mar 2021 …

Chiesi’s Bronchitol (mannitol) Inhalation Powder Receives the US FDA’s Approval for Cystic Fibrosis Read More »

Lucira’s All-In-One Test Kit Receives the US FDA’s EUA as the First COVID-19 Test for Self-Testing at Home

Shots: The Lucira’s COVID-19 all-in-one test kit test has been authorized for home use with self-collected nasal swab samples in individuals aged≥ 14yrs. who are suspected of COVID-19 by their HCPs It is also authorized for use in POC settings for all ages, but samples must be collected by an HCP when the test is …

Lucira’s All-In-One Test Kit Receives the US FDA’s EUA as the First COVID-19 Test for Self-Testing at Home Read More »

Sanofi’s Avalglucosidase alfa Receives US FDA’s Priority Review as Enzyme Replacement Therapy for Pompe Disease

Shots: The US FDA accepted for priority review the BLA of avalglucosidase alfa for long-term enzyme replacement therapy for the treatment of patients with Pompe disease. The target action date for the FDA decision is May 18, 2021 The BLA is based on positive data from two trials: P-III COMET study and P-II mini-COMET study …

Sanofi’s Avalglucosidase alfa Receives US FDA’s Priority Review as Enzyme Replacement Therapy for Pompe Disease Read More »

ViiV Healthcare’s Cabotegravir Receives US FDA’s Breakthrough Therapy Designation for HIV Prevention

Shots: This BT designation is based on P-IIb/III HPTN 083 study assessing cabotegravir (q8w) vs. FTC/TDF tablets (qd PO, 200/300 mg) in a study population of 4,566 for HIV prevention. The data were presented at the 23rd International AIDS Conference (AIDS 2020) Results: HPTN 083 study showed that cabotegravir was 66% more effective at preventing …

ViiV Healthcare’s Cabotegravir Receives US FDA’s Breakthrough Therapy Designation for HIV Prevention Read More »

NightWare Receives the US FDA’s Approval for the First Medical Device to Stop NightMAres Related to PTSD

Shots: The US FDA has permitted marketing of a device intended for the temporary reduction of sleep disturbance related to nightmares in patients aged ≥22 yrs. suffering from nightmare disorder or PTSD Nightware utilizes Apple Watch and an iPhone that is configured and logged into a software application and the Nightware server and monitors the …

NightWare Receives the US FDA’s Approval for the First Medical Device to Stop NightMAres Related to PTSD Read More »

Bayer Reports Regulatory Submission of Finerenone (BAY 94-8862) to the US and EU for CKD and T2D

Shots: The MAA submitted to the EMA and the NDA to the US is based on P-III FIDELIO-DKD study assessing finerenone (10/20mg, qd) + SOC vs PBO in ~5,700 patients with CKD and T2D Result: reduction in the combined risk of time to kidney failure; a decrease of eGFR (≥ 40%) from baseline @4wks. or …

Bayer Reports Regulatory Submission of Finerenone (BAY 94-8862) to the US and EU for CKD and T2D Read More »

Lilly’s Bamlanivimab (LY-CoV555) Receives the US FDA’s EUA for the Treatment of Recently Diagnosed COVID-19

Shots: The EUA is based on P-II BLAZE-1 study assessing bamlanivimab (700/2800/7000 mg) as monothx. vs PBO or in combination with second Ab for the treatment of symptomatic COVID-19 in the outpatient setting. The EUA is granted for bamlanivimab (LY-CoV555, 700 mg) Patients showed reduced viral load & rates of symptoms & hospitalization. The EUA …

Lilly’s Bamlanivimab (LY-CoV555) Receives the US FDA’s EUA for the Treatment of Recently Diagnosed COVID-19 Read More »

The US FDA’s Advisory Panel Slams Biogen’s Aducanumab for Alzheimer’s Disease

Shots: The advisory committee voted 1-8, with 2 members voting uncertain that EMERGE study (without regard for ENGAGE study) provides strong evidence, supporting the effectiveness of aducanumab as a treatment for AD. The committee also voted 0-7 with 4 members voting uncertain that PRIME study provides supporting evidence for aducanumab The committee voted 5-0 and …

The US FDA’s Advisory Panel Slams Biogen’s Aducanumab for Alzheimer’s Disease Read More »

AstraZeneca’s Brilinta (ticagrelor) Receives the US FDA’s Approval to Reduce the Risk of Stroke in Patients with an Acute Ischemic Stroke or High-Risk Transient Ischemic Attack

Shots: The approval is based on P-III THALES study involves assessing Brilinta (180mg as a loading dose followed by 90mg, bid) + aspirin vs aspirin as monothx. in 11,000 patients with non-cardioembolic acute ischaemic stroke or high-risk TIA for 30days Results demonstrated a reduction in the rate of the composite 1EP of stroke & death …

AstraZeneca’s Brilinta (ticagrelor) Receives the US FDA’s Approval to Reduce the Risk of Stroke in Patients with an Acute Ischemic Stroke or High-Risk Transient Ischemic Attack Read More »

Boston Scientific’s Ranger DCB Receives the US FDA’s Approval for Peripheral Artery Disease in the SFA and PPA

Shots: The approval is based on the RANGER II SFA pivotal study assessing the Ranger DCB vs standard PTA for the treatment in patients with PAD in the SFA & PPA The study met its both 1EPs @12mos. i.e. MAE (94.1% vs 83.5%); lesion revascularization rate (5.5% vs 16.5%); Binary primary patency (82.9% vs 66.3%); …

Boston Scientific’s Ranger DCB Receives the US FDA’s Approval for Peripheral Artery Disease in the SFA and PPA Read More »

Janssen Reports sNDA Submission of Paliperidone Palmitate 6-Month (PP6M) to the US FDA for Schizophrenia

Shots: The sNDA submission is based on P-lll Route-6 study assessing PP6M vs PP3M in 702 patients with schizophrenia previously stabilized on corresponding doses of PP1M or PP3M across 20 countries Results demonstrated non-inferior efficacy of PP6M to PP3M on the 1EPs of time to relapse at the end of the 12mos. period in both …

Janssen Reports sNDA Submission of Paliperidone Palmitate 6-Month (PP6M) to the US FDA for Schizophrenia Read More »

PharmaShots’ Key Highlights of Third Quarter 2020

The third quarter of 2020 was flooded with major acquisitions in the pharma and biotech industry along with multiple approvals. Starting with the latest acquisitions, Gilead acquired Immunomedics for ~$21B, Illumina acquired GRAIL for ~$8B, J&J acquired Momenta Pharmaceuticals for $6.5B and Nestlé acquired Aimmune Therapeutics for $2.6B, Sanofi acquired Principia Biopharma for ~$3.68B  Apart …

PharmaShots’ Key Highlights of Third Quarter 2020 Read More »

Medtronic’s Abre Venous Stent Receives the US FDA’s Approval to Treat Venous Outflow Obstruction

Shots: The approval is based on ABRE clinical study assessing the Abre stent in 200 patients with iliofemoral venous outflow obstruction across the spectrum of deep venous obstruction including those with the post-thrombotic syndrome, NIVL & those who presented with an aDVT. The study also includes a challenging patient population, 44% of whom required stents …

Medtronic’s Abre Venous Stent Receives the US FDA’s Approval to Treat Venous Outflow Obstruction Read More »

Polarean Reports the NDA Submission of Hyperpolarised 129Xenon Gas Drug-Device Diagnostic to the US FDA for Lung Imaging

Shots:   The company reports NDA submission and request for PR to the US FDA for hyperpolarised 129Xenon gas used to evaluate the pulmonary function and to visualize the lung using MRI. The NDA submission follows the completion of two P-III studies, demonstrating the effective measurement of regional lung ventilation Both the studies met their 1EPs …

Polarean Reports the NDA Submission of Hyperpolarised 129Xenon Gas Drug-Device Diagnostic to the US FDA for Lung Imaging Read More »

MorphoSys Reports Submission of Tafasitamab’s BLA to the US FDA to Treat Relapsed or Refractory Diffuse Large B Cell Lymphoma (r/r DLBCL)

Shots: The BLA submission is based on L-MIND P-II trial data results assessing Tafasitamab + lenalidomide in patients with r/r DLBCL and retrospective observational matched control cohort Re-MIND evaluating efficacy outcomes of r/r DLBCL patients who received Tafasitamab + lenalidomide vs lenalidomide monothx Re-MIND has met its 1EPs and has shown improved ORR of the …

MorphoSys Reports Submission of Tafasitamab’s BLA to the US FDA to Treat Relapsed or Refractory Diffuse Large B Cell Lymphoma (r/r DLBCL) Read More »