Biologics - FDA Webview

Biologics

Imfinzi sBLA Gets Priority Review for Bladder Cancer

FDA has accepted for priority review an AstraZeneca supplemental BLA for Imfinzi (durvalumab) in combination with enfortumab vedotin (EV) for treating patients with muscle-invasive bladder cancer (MIBC) who are ineligible for or have declined cisplatin-based chemotherapy. The agency set a user fee review action target date for upcoming

Biologics

Are Comparative Clinical Studies of Biologics Worth the Time and Cost?

FDA says a meta-analysis conducted by CDER scientists found that pharmacokinetic similarity studies provided useful information to descriptively evaluate the immunogenicity and safety of biologics, and that comparative clinical studies did not appear to be more definitive. In an online post, the agency says the scientists suggest that their

Biologics

CBER Sponsor Regulatory Meetings SOPP

CBER has issued a Standard Operating Policy and Procedure (SOPP) as a guide for Center staff in scheduling and conducting regulatory meetings between individuals in the Center and representatives of regulated companies and/or individual sponsor-investigators to address issues relating to product development. The SOPP applies to meetings for

Biologics

FDA Wants New Survival Trial Before Compass Files Tovecimig BLA

FDA has recommended that Compass Therapeutics conduct an additional clinical trial demonstrating an overall survival benefit before submitting a BLA for tovecimig and its use in previously treated, advanced biliary tract cancer patients, despite positive response rate and progression-free survival results from the Phase 2/3 COMPANION-002 study,

Biologics

Ultragenyx Gains Approval for Resubmitted Gene Therapy

Biologics

Interchangeable Product Development Strategy Outlined

Biologics

FDA Clears Path for Rocket to Complete Pivotal Gene Therapy Trial

Biologics

Scholar Rock’s Spinal Muscular Atrophy Therapy Approved

Biologics

Nanoscope BLA Accepted for Vision Restoration Gene Therapy

FDA has accepted for review a Nanoscope Therapeutics BLA seeking approval for Mogenry (sonpiretigene isteparvovec, MCO-010), an optogenetic gene therapy designed for vision restoration in patients with retinitis pigmentosa (RP) having severe vision loss. “The strength of our RESTORE data, together with the durability we have observed through long-

Biologics

uniQure Files BLA for Huntington’s Gene Therapy

uniQure has submitted a BLA seeking accelerated approval of ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for treating Huntington’s disease. The company’s submission requested a priority review. The filing is based on a three-year data analysis from a Phase I/II clinical study of ifezuntirgene inilparvovec,

Biologics

FDA Approves Updated Covid-19 Vaccines for Upcoming Season

FDA has approved updated Covid-19 vaccines from Moderna, Pfizer-BioNTech and Novavax/Sanofi for the upcoming vaccination season. The vaccines are formulated to better match the XFG variant currently in circulation. The updated vaccines include Moderna's Spikevax and mNEXSPIKE, Pfizer-BioNTech's Comirnaty and Novavax-Sanofi&

Biologics

FDA Approves First Drug for Warm Autoimmune Hemolytic Anemia

FDA has approved a Janssen Biotech BLA for Imaavy (nipocalimab-aahu) injection for warm autoimmune hemolytic anemia (wAIHA) in adult and pediatric patients 12 years of age and older currently or previously treated with corticosteroids. The rare blood disorder causes a patient’s immune system to mistakenly attack and destroy

Biologics

R3 Selling Unapproved Drugs, Biologics, FDA Says

An 11/17-12/16/2025 FDA inspection at the R3 Medical Companies’ Bello Bio and Regen Suppliers units in Scottsdale, AZ, documented that Bello contract manufactures and labels several umbilical cord-derived products and Regen approves, labels, stores, and distributes the Bello products and an umbilical cord product contract

Biologics

Jemperli sBLA for Rectal Cancer Accepted for Priority Review

FDA has accepted for priority review a GSK supplemental BLA for Jemperli (dostarlimab) for patients with previously untreated Stage II and III mismatch repair deficient/microsatellite instability-high locally advanced rectal cancer. The agency has set a user fee review action target date of 2/2027, according to the company,

Biologics

HHS Seeks Input on Categories of Federal Vaccine Recommendations

HHS is seeking public input on how the federal government classifies vaccine recommendations, including whether its current system gives patients and parents enough choice and clearly reflects the strength of scientific evidence. A new Federal Register notice says the Department is reviewing three existing categories: routine recommendations, which generally apply

Biologics

Clinical Hold on Regenxbio Hunter Syndrome Gene Therapy

FDA has placed a clinical hold on Regenxbio's investigational gene therapy RGX-121 after asymptomatic spine MRI findings were identified in five participants in a clinical trial evaluating the treatment for Hunter Syndrome or Mucopolysaccharidosis Type II (MPS II). In response, the company says it does not expect

Biologics

FDA 3-Month Review Extension on Capricor Gene Therapy

FDA has extended by three months its review of a Capricor Therapeutics BLA for Deramiocel, an investigational cell therapy for Duchenne muscular dystrophy (DMD). The new user fee review action target date is 11/22. “As part of its ongoing discussions with the FDA following the July 2026 Advisory Committee

Biologics

CBER Updates Process for Formal Disputes With Sponsors

CBER has updated its standard operating procedures for resolving scientific/procedural disputes between sponsors and the Center, establishing revised procedures and adding medical devices to the formal dispute resolution process. The updated SOPP 8005: Formal Dispute Resolution Process applies to disputes between sponsors and CBER review staff involving regulatory submissions.

Biologics

Cellular, Gene Therapy Product FAQs

FDA has published a guidance, Frequently Asked Questions—Developing Potential Cellular and Gene Therapy Products, to help facilitate the development of safe, effective, and high-quality cellular and gene therapy (CGT) products. The document contains common questions directed to FDA and spans multiple disciplines, including regulatory review; chemistry, manufacturing, and

Biologics

Draft Guidance on Potency Testing for Active Immunotherapy Products

FDA has issued a draft guidance that outlines recommendations for developing potency assays for active immunotherapy products (ACTIMPs), including personalized cancer immunotherapies, vectored products and cell-based therapies. The document, “Potency Assessment of Active Immunotherapy Products,” is intended to provide a science- and risk-based framework for evaluating whether ACTIMPs

Biologics

Senators Release 2021 Woodcock Email on Covid-19 Concern

Two Republican senators have released a 5/2021 email from then-acting FDA commissioner Janet Woodcock in which she raised concerns about reports of unexplained adverse events that appeared following Covid-19 vaccination and urged federal health officials to consider conducting a study. Sens. Ron Johnson (R-WI) and Rand

Biologics

Capricor Plans Deramiocel BLA Amendment After FDA Input

Capricor Therapeutics says it plans to amend its BLA for deramiocel to modify its proposed indication after discussions with FDA produced a potential path toward approval of the Duchenne muscular dystrophy therapy based on its Phase 3 trial’s primary endpoint. FDA has indicated it is willing to review an