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
enforcement
FDA's anticipated proposal to restrict direct-to-consumer (DTC) prescription drug advertising could face significant statutory and First Amendment challenges, while changes in agency leadership raise questions about whether the administration's advertising enforcement campaign will maintain its momentum, attorneys said during a recent FDLI conference panel.
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,
enforcement
FDA has released 33 additional Form FDA-483 inspection reports on 10/30, covering drug and biologic manufacturers, medical device companies, and human tissue establishments in the U.S. and overseas. The newly disclosed records include inspections of Baxter Healthcare, Catalent Maryland, Andelyn Biosciences, Charles River Laboratories, Autolus, AGC Biologics,
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
Drugs
FDA has placed a partial clinical hold on Bright Minds Biosciences' planned Phase 2 trial of BMB-101 in patients with developmental and epileptic encephalopathies (DEEs), citing toxicity findings in mice and insufficient pharmacokinetic and juvenile toxicity data to support pediatric enrollment. The proposed U.S. trial is 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
Drugs
FDA has extended by three months its review of an Agios Pharmaceuticals supplemental NDA for mitapivat, an oral pyruvate kinase (PK) activator, for treating sickle cell disease. The user fee review action target date is now 2/1/2027. The company says FDA recently requested additional information to evaluate whether
Devices
Drugs
FDA has approved revised labeling for three additional estradiol transdermal patch products, removing boxed-warning statements about cardiovascular disease, breast cancer and probable dementia, HHS announced 10/30, prompting renewed criticism over the agency's decision to proceed without a formal advisory committee meeting or publicly presenting detailed scientific
FDA Policy/General
The American Medical Association (AMA) is urging FDA to “uphold rigorous, evidence-based standards for evaluating botanical drug products,” warning that products marketed with unproven health claims can pose serious risks when their safety and efficacy have not been established. In comments to FDA, the AMA emphasized that botanical products
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
Drugs
CDER’s Office of Prescription Drug Promotion (OPDP) issued 30 enforcement letters during fiscal year 2026, including 28 untitled letters and two warning letters, according to office director Twyla Mosey, who told a Food and Drug Law Institute conference that many promotional violations might have been avoided if drugmakers had
Drugs
FDA has posted an International Council for Harmonization revised final guidance to clarify how drug developers should evaluate the potential for investigational drugs to prolong the QT/QTc interval and cause potentially serious cardiac arrhythmias, including circumstances in which traditional clinical cardiac safety studies may not be feasible. The guidance,
Drugs
Former FDA principal deputy commissioner, acting commissioner, and CDER director Janet Woodcock says reforms are needed to make individualized gene therapies a viable development opportunity for rare diseases. Writing in a BioCentury online commentary, Woodcock says FDA already has the statutory authority to address several of the barriers preventing the
Drugs
Vanda Pharmaceuticals and FDA are asking a federal judge to resolve a dispute over the agency's partial clinical hold on a yearlong trial of tradipitant for gastroparesis, with the company arguing that FDA's insistence on additional animal toxicity testing violates the FDA Modernization Act 2.0
Devices
FDA has issued a revised a 6/2024 draft guidance outlining the performance characteristics manufacturers should evaluate, validate and control for medical devices used to deliver drugs and biological products, including drug-device combination products. The guidance, Essential Drug Delivery Performance Characteristics for Devices Intended to Deliver Drugs and Biological
Devices
FDA has issued a Warning Letter to Utah-based Chirp Innovations LLC for promoting its cleared Halo Wireless Muscle Stimulator for menstrual pain without obtaining additional marketing clearance and for widespread deficiencies in its quality system and medical device reporting procedures. The 10/2 Warning Letter followed an 11/2025
Drugs
FDA has accepted for review a resubmitted NDA for Izipry (naloxone nasal powder), a high-dose naloxone rescue medication for opioid overdose. The agency has set a user fee review action target date of 3/29/2027. Izipry is being developed using Orexo’s proprietary AmorphOX powder-based drug delivery
Drugs
FDA has published an updated Concept of Operations outlining how CDER and the Office of Inspections and Investigations (OII) (formerly the Office of Regulatory Affairs) “collaborate in a coordinated, programmatically aligned environment regarding marketing application assessment, inspections, and related activities.” The document covers preapproval, postapproval, surveillance, and for-cause inspection
enforcement
Medical device manufacturers increasingly using artificial intelligence (AI) for quality assurance and regulatory functions must maintain human oversight of those systems, according to former senior FDA officials who say the agency is also beginning to incorporate AI into its inspection and postmarket surveillance activities. Speaking on the 10/26 FDA
Drugs
A Duke Margolis Institute for Health Policy white paper, “From Research to Care: Modernizing U.S. Clinical Studies for Cures Into Care,” lists several priority actions that it says private organizations and collaborations need to take to reform the U.S. fragmented clinical research system. The report reviews the current