Biologics
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 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 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
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
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 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
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 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 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
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
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
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 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
Biologics
FDA has approved a Moderna BLA for mFLUSIVA (mRNA-1010), a new influenza vaccine for use in all adults 50 years and older. The approval follows a June Vaccines and Related Biological Products Advisory Committee meeting that supported the vaccine’s approval. "The FDA approval of mFLUSIVA, our fourth
Biologics
FDA has granted accelerated approval to Replimune for its Tudriqev (vusolimogene oderparepvec-wtpg), a genetically modified oncolytic viral therapy, in combination with Bristol Myers Squibb's Opdivo (nivolumab), for treating adult patients with unresectable advanced cutaneous melanoma who experienced disease progression on a programmed death receptor-1 (PD-1)
Biologics
FDA has accepted for review an Allergan Aesthetics supplemental BLA for Botox Cosmetic (onabotulinumtoxinA) for the temporary improvement in the appearance of marked to very marked masseter muscle prominence (MMP) associated with masseter muscle activity in adults. “MMP can contribute to the appearance of a wider or more square lower
Biologics
A new FDA draft guidance is outlining how sponsors should develop and evaluate container closure systems and device components used with biosimilar and interchangeable biosimilar products, providing a clearer framework for demonstrating that design differences from reference products do not compromise safety or effectiveness. The document focuses on therapeutic protein
Biologics
Sen. Ron Johnson (R-WI), chairman of the Senate Permanent Subcommittee on Investigations, has requested that former CBER director Peter Marks, former CDER officials Narayan Nair, Steven Anderson, and Manette Niu, and former National Institute of Allergy and Infectious Diseases director Anthony Fauci appear for transcribed interviews about their roles
Biologics
FDA has accepted for priority review a Johnson & Johnson supplemental BLA for subcutaneous Rybrevant Faspro (amivantamab and hyaluronidase-lpuj) for adults with recurrent or metastatic head and neck squamous cell carcinoma. If approved, it would provide a new treatment for patients whose disease has progressed following platinum-based chemotherapy
Biologics
A 5/5 FDA guidance on chemistry, manufacturing, and controls flexibilities for developing human cellular and gene therapy (CGT) products is changing the landscape of regulatory approval for such products. A BioProcess International online column analyzing the guidance, which is being implemented immediately, says FDA has abandoned its long-standing
Biologics
FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee has voted 10 to 3 to recommend approval of Replimune's experimental oncolytic immunotherapy RP1 (vusolimogene oderparepvec) in combination with Bristol Myers Squibb's Opdivo for patients with advanced melanoma, despite FDA reviewers' conclusions that the application lacked
Biologics
An FDA advisory committee voted 9 to 3 against recommending approval for Capricor Therapeutics' investigational cell therapy deramiocel, for treating cardiomyopathy associated with Duchenne muscular dystrophy (DMD), dealing a significant setback to the company's approval bid ahead of an 8/22 user fee review decision target date.