Biologics
FDA is investigating the safety of Novartis’ Zolgensma, an adeno-associated virus (AAV) vector-based gene therapy for spinal muscular atrophy (SMA), based on postmarketing adverse event reports of malignancies in treated patients. The adverse events include confirmed reports of “product-related insertional oncogenesis involving the GLI1 (Glioma-Associated Oncogene
Biologics
Chemistry, manufacturing and controls (CMC) concerns accounted for 58% of the topics raised in FDA information requests during reviews of seven adeno-associated virus (AAV) gene therapy applications, according to an analysis by agency scientists published 11/6 in Nature Reviews Drug Discovery. The analysis, conducted by CBER researchers, examined
Drugs
FDA's drug and biologics review centers added a net 84 employees during the fourth quarter of fiscal year 2026, reversing earlier staffing losses but leaving their combined workforce about 18% below its level at the beginning of fiscal 2025, according to newly updated agency hiring data. The figures
Biologics
Novartis has disclosed nine reports of tumors in children treated with its spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec-xioi), including two cases in which investigators identified evidence of viral vector DNA integration into the patients' genomes, raising new questions about the treatment's long-term
Biologics
FDA has issued final guidance recommending a comprehensive, risk-based approach to assuring the potency of cellular and gene therapy (CGT) products throughout development and commercial manufacturing, replacing its January 2011 guidance focused on potency testing. The guidance, Potency Assurance for Cellular and Gene Therapy Products, recommends that manufacturers integrate
Biologics
FDA's developing rare disease regulatory framework could make early patient engagement an essential part of drug development, rather than an activity primarily associated with advisory committee meetings and later stages of regulatory review, according to CBER director Karim Mikhail. Speaking at a 10/16 Reagan-Udall Foundation meeting,
Biologics
FDA has accepted for review a UniQure BLA seeking accelerated approval of ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for treating Huntington’s disease. FDA told the company that it is tentatively planning to discuss this application at a future advisory committee meeting. The filing is based on a
Biologics
Gilead Sciences says two clinical trials of its investigational CAR-T cell therapy KITE-753 have been placed on partial clinical hold following reports of serious neurological adverse events in a small number of patients. The company disclosed the regulatory action during its recent third-quarter earnings call. The affected
Biologics
CBER’s Advertising and Promotional Labeling Branch (APLB) is warning biologic marketers against using progression-free survival and overall survival data from single-arm studies to support promotional claims that cannot be substantiated by the underlying clinical evidence. Speaking at a 10/28 Food and Drug Law Institute advertising and
Biologics
FDA has posted a draft guidance outlining a regulatory framework that would allow manufacturers of individualized genome-editing (GE) therapies to include multiple patient-specific treatment variants in a single BLA and add additional variants after approval without necessarily conducting new clinical trials. The draft guidance, Individualized Human Genome Editing
Biologics
FDA has accepted for priority review a Merck BLA for sacituzumab tirumotecan (sac-TMT), an investigational TROP2-directed antibody-drug conjugate (ADC), for adults with endometrial cancer previously treated with platinum-based chemotherapy and a PD-1 or PD-L1 inhibitor, administered separately or in combination. The agency has set
Biologics
FDA has approved AbbVie’s subcutaneous (SC) induction regimen for Skyrizi (risankizumab-rzaa) for adults with moderately to severely active Crohn's disease (CD). “Patients now have the option to start SKYRIZI treatment at home, with induction doses delivered by an on-body injector (OBI) induction pack and then
Biologics
Bristol Myers Squibb reported preliminary safety and efficacy data from a Phase 1 study of its investigational CAR T-cell therapy zolacabtagene autoleucel (zola-cel; BMS-986353) in patients with multiple sclerosis, but disclosed that one trial participant died of septic pneumonia 44 days after receiving treatment. The death reportedly
Biologics
If vaccine advocates gain substantial support in Congress following next month’s midterm elections, the Trump administration could still accelerate changes to federal vaccine policies in 2027, potentially limiting the effectiveness of congressional oversight, two former federal health policy officials warn. In a 10/20 MedPage Today commentary, Steven Grossman,
Biologics
Merck and Moderna are reporting positive recurrence-free survival (RFS) and distant metastasis-free survival (DMFS) results from a highly anticipated Phase 3 trial evaluating their personalized cancer vaccine, intismeran autogene, in combination with Keytruda (pembrolizumab). The Phase 3 INTerpath-001 trial is evaluating intismeran autogene, an investigational mRNA-based
Biologics
A Georgia federal court has determined that FDA misread its own regulations in determining that a MiMedx wound care powder product was not a human cell, tissue, or cellular- or tissue-based product (HCT/P) because it was not minimally manipulated. Attorneys Vanessa Hampton and Alan Minsk (Arnall Golden Gregory)
Biologics
Richard Forshee, an FDA vaccine safety official demoted during former CBER director Vinay Prasad's tenure, has returned to a senior leadership position at the Center. Forshee announced in a 10/19 LinkedIn post that he had become director of CBER's Office of Biostatistics and Pharmacovigilance, which
Biologics
FDA has granted GSK accelerated approval for a new use of the immunotherapy Jemperli (dostarlimab-gxly) —treating adults with previously untreated stage II or stage III locally advanced rectal cancer. “At these stages, the cancer has grown into nearby tissue or reached nearby lymph nodes but has not spread to
Biologics
FDA should eliminate “redundant biosimilar trials when rigorous scientific evidence already establishes similarity,” a 10/16 Newsweek opinion piece by Sarfaraz K. Niazi says. The commentary argues that advances in analytical testing now allow manufacturers to demonstrate that proposed biosimilars are highly similar to reference biologics without necessarily conducting additional
Biologics
FDA has accepted for review a Pierre Fabre Pharmaceuticals BLA resubmission with a proposed indication as monotherapy for treating adult and pediatric patients 2 years of age and older with Epstein-Barr virus-positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. The agency
Biologics
FDA has approved Altor BioScience’s Anktiva (nogapendekin alfa inbakicept-pmln) with Bacillus Calmette-Guérin (BCG) for adults with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). The approval revises the prior indication for adults with BCG-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumors to
Biologics
FDA has accepted for review a Formycon/Zydus Lifesciences BLA for FYB206, a biosimilar to Merck’s blockbuster cancer drug Keytruda (pembrolizumab). A statement says the biosimilar was developed without a comparative Phase 3 efficacy trial, following a development pathway agreed upon with FDA in early 2025. The companies say