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
As FDA moves to reduce animal testing requirements for certain drugs, researchers say emerging technologies involving human organoids, organ-on-a-chip systems and artificial intelligence could replace some traditional animal studies, although significant scientific and regulatory limitations remain. In a Perspective published 11/2 in The Innovation, researchers from
FDA Policy/General
FDA officials remain confident that Congress will reauthorize the agency's four major user fee programs before they expire next September, although Democratic control of Congress and the prospect of Sen. Bernie Sanders (I-VT) chairing the Senate HELP Committee could complicate the reauthorization over the already-negotiated agreements.
Drugs
Merck's investigational GLP-1/glucagon receptor dual agonist efinopegdutide (MK-6024) demonstrated higher rates of MASH resolution and liver fibrosis improvement than placebo in a Phase 2b trial involving patients with metabolic dysfunction-associated steatohepatitis (MASH) and moderate to advanced fibrosis. The results, presented at the American Association
Drugs
FDA officials say drug manufacturers may be able to establish dosing recommendations and characterize drug-drug interaction risks without conducting additional clinical studies, including through pharmacokinetic modeling and real-world data, provided the evidence is scientifically justified. During a series of FDA presentations released 11/4 on [Drug Interaction Information
Drugs
FDA has granted Janssen Biotech accelerated approval for Rybrevant Faspro (amivantamab and hyaluronidase-lpuj) and its use in treating adults with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) whose disease has progressed on or after platinum-based chemotherapy and immunotherapy. “HNSCC is an aggressive, life-threatening disease
Drugs
FDA associate commissioner for inspections and investigations Elizabeth Miller says unannounced drug inspections in China and India increased significantly in FY 2026 compared with FY 2025 inspection numbers. As reported by the Regulatory Affairs Professional Society’s online Regulatory Focus newsletter, she spoke at the Association for Accessible Medicines’ GRx+
Drugs
Yale School of Medicine researchers say there are steps FDA could take to improve the evaluation of elements in Risk Evaluation and Mitigation Strategies (REMS) programs. Writing in JAMA Internal Medicine, the researchers say REMS requirements intended to increase patient and prescriber knowledge of medication safety and risks include medication
enforcement
FDA 11/5 posted five additional Form FDA-483 inspection reports, including four involving manufacturers in Japan and one involving a U.S. drug outsourcing facility. The newly published records cover inspections conducted between October 2023 and September 2026. They include: * LeeSar Inc., Florida: Inspection dated September 23, 2026, involving
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
Independent news and analysis of FDA regulation, policy and enforcement
Commenting on an Ascendis Pharma 10/6 petition asking FDA to require additional information about BridgeBio’s infigratinib to resolve safety questions involving its potential indication to treat children with achondroplasia, Center for Medicine in the Public Interest president Peter Pitts says FDA should be held to the same scientific
VivoSim Labs executive chairman Keith Murphy says that now that FDA has finalized a rule permitting non-animal methods for drug safety testing, it should move quickly to make a determination that some non-animal methods have already reached a well-qualified state that makes them supportive of the agency’
FDA has accepted for review an Elevar Therapeutics and Hengrui Pharmaceuticals resubmission for rivoceranib plus camrelizumab as a first-line treatment for patients with unresectable or metastatic hepatocellular carcinoma. The agency has set a user fee review action date of 4/9/2027. On 10/9, Elevar resubmitted the NDA
FDA is planning to hold a two-day public workshop 12/2-3 to examine the benefits and risks of robotic medical devices capable of autonomous operation or remote teleoperation, potentially informing future regulatory policy for the emerging technologies. The CDRH hybrid workshop is intended to bring together device manufacturers,
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
The Pharmaceutical Research and Manufacturers of America (PhRMA) has asked a federal court to invalidate the Trump administration's Medicare international drug-pricing rule, arguing that CMS lacks statutory authority to impose foreign-reference pricing on drugs covered under Medicare Part B. In an 11/4 motion for summary
Democrats flipping both chambers of Congress in the 11/3 midterm elections has complicated the confirmation prospects of President Trump's nominee to lead FDA, Heidi Overton, while setting the stage for increased congressional scrutiny of the agency's drug approval decisions, vaccine policies and regulatory independence. The
FDA has issued Vanda Pharmaceuticals an untitled letter citing a television advertisement for its motion sickness drug Nereus (tradipitant), alleging that a high-speed car chase and other dramatic scenes misleadingly portray the drug's effectiveness and safety while interfering with viewers' understanding of its risks. The agency
In its latest five-year report, covering 7/1/2020 to 6/30/2025, FDA says the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) have “fundamentally transformed the landscape of pediatric drug development, delivering substantial benefits to children and families across the United States
FDA referenced artificial intelligence in just seven of 3,011 Warning Letters issued between 1/2022 and 9/28/2026, with only one letter explicitly identifying a company's use of AI as a regulatory violation, according to a new study published in Discover Pharmaceutical Sciences. The retrospective analysis,
President Trump 11/3 signed into law legislation extending federal programs supporting access to investigational amyotrophic lateral sclerosis (ALS) treatments through fiscal 2031 and requiring FDA to develop a new five-year action plan for rare neurodegenerative diseases. The bipartisan Accelerating Access to Critical Therapies for ALS Reauthorization Act of
International researchers say their review of 26 drugs for 35 pediatric indications given accelerated approval by FDA between 2000 and 2020 suggests there are persistent challenges in generating timely, high-quality pediatric evidence under the accelerated approval pathway and emphasizes the need for innovative data-driven strategies to strengthen confirmatory