Drugs
Drug sponsors are not required to submit model analysis plans in advance under the new ICH M15 guideline on model-informed drug development (MIDD), and models that fail to meet predefined validation criteria are not necessarily disqualified from supporting regulatory decisions, international regulatory experts said during an 10/29 FDA-
Drugs
AstraZeneca has petitioned FDA to withhold approval of a Cipla ANDA for proposed new strengths of generic Lynparza (olaparib) tablets unless the generic company provides additional evidence addressing formulation differences, tablet size and potential medication errors. Lynparza, a PARP inhibitor approved for certain ovarian, breast, pancreatic and prostate cancers, is
enforcement
FDA's stepped-up enforcement against prescription drug advertising may be having an unintended consequence: Some pharmaceutical companies are becoming more willing to accept promotional risks because the agency has issued so many enforcement letters without consistently escalating to more serious actions, attorneys said during a recent Food and
FDA Policy/General
FDA's use of advisory committees has declined since 2010, while recent research suggests financial conflicts of interest among committee members have increased after an earlier decline, according to a systematic review published in the 10/29 JAMA Network Open. Researchers from Yale University and other institutions examined 146
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
Independent news and analysis of FDA regulation, policy and enforcement
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
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
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
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
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
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
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,
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
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
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
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