Biologics
FDA has accepted for priority review a BioMarin Pharmaceutical supplemental BLA for Palynziq (pegvaliase-pqpz) to expand the therapy’s use for treating adolescents aged 12–17 with phenylketonuria (PKU). The agency has set a user fee review action target date of 2/28/2026. The submission is supported by
Drugs
A new Friends of Cancer Research white paper, “Seamless Clinical Trial Designs in Rare Cancers: Leveraging Operational and Adaptive Strategies to Accelerate Drug Development,” concludes that when thoughtfully designed, such trials can accelerate timelines, reduce redundancy, and generate robust evidence from limited populations without compromising scientific integrity. The paper is
Biologics
FDA is proposing a major shift in how biosimilars are evaluated, signaling that many products may no longer need traditional comparative clinical efficacy studies (CES) to demonstrate biosimilarity. A new draft guidance, Scientific Considerations in Demonstrating Biosimilarity to a Reference Product: Updated Recommendations for Assessing the Need for Comparative Efficacy
Drugs
FDA has granted Revolution Medicines an orphan drug designation for its investigational therapy daraxonrasib (also known as RMC-6236) for treating pancreatic cancer. Daraxonrasib is currently being evaluated in a global Phase 3 study (RASolute 302) in patients with second-line metastatic pancreatic ductal adenocarcinoma (PDAC). The company says it
Biologics
FDA has issued Regeneron a complete response letter for its pre-filled Eylea HD syringe supplemental BLA due to unresolved inspection findings at third-party manufacturer Catalent’s troubled Indiana facility. Regeneron now says it plans to submit by 1/2026 an application to include a new pre-filled syringe
Devices
FDA inspections at three Royal Philips medical device manufacturing facilities — Bothwell, WA, Reedsville, PA, and Eindhoven, Netherlands — identified Quality System Regulation and other violations in the firm’s manufacturing of several medical devices. A just-released 9/9 Warning Letter says the specific violations were: * failure to establish and maintain
Drugs
Unicycive says that after discussions with FDA and the company’s third-party manufacturing vendor, it plans to resubmit its NDA for oxylanthanum carbonate (OLC) to FDA before the end of 2025. FDA had issued a 6/30 complete response letter (CRL) citing concerns about the compliance status of Unicycive’
Drugs
A 4/22-4/25 FDA inspection at the Tranzonic Innocore Sales & Marketing drug manufacturing facility in Woodstock, Ontario, Canada, found four repeat Current Good Manufacturing Practice (CGMP) violations in the firm’s production of finished drugs. A 10/16 Warning Letter says the specific violations were: * failure to
Drugs
Acting Office of New Drugs (OND) director Mary Tran Thanh-Hai has been officially named the new OND director, replacing previous head Peter Stein who was forced out of the agency under mass layoffs in April. At the time, Stein was offered a reassignment to a position in patient affairs,
Devices
Warning Letters sent recently by FDA to two medical device companies instructed the firms to respond within 30 days, rather than the typical 15 days. Three Hyman, Phelps & McNamara attorneys write in their firm’s FDA Law Blog that their review of some 200 Warning Letters issued by CDRH
Devices
FDA has published a draft guidance, Menstrual Products—Performance Testing and Labeling Recommendations, with the agency’s recommendations for performance testing and labeling of such products, and also information for inclusion in 510 (k) submissions, when necessary, for certain menstrual products. The document says its recommendations apply to tampons, pads,
Drugs
FDA has posted a seven-item Form FDA-483 after inspecting Dr. Reddy’s Mirfield, West Yorkshire, UK, active pharmaceutical ingredient (API) manufacturing facility 9/1-5/2025. The key GMP deficiencies noted were: * Failure to establish adequate specifications, sampling plans, and testing procedures to ensure that raw material and
Drugs
Intellia Therapeutics has paused patient dosing and screening for its MAGNITUDE and MAGNITUDE-2 Phase 3 trials of nex-z, a CRISPR-based therapy for transthyretin amyloidosis with cardiomyopathy and polyneuropathy, due to a recent patient injury. The decision follows a 10/24 report on a patient injury in the
Drugs
Senate Special Committee on Aging leaders are demanding answers from HHS on what they describe as serious national security and public health risks tied to America’s dependence on foreign pharmaceutical supply chains. In a 10/24 letter to secretary Robert F. Kennedy Jr., committee chair Rick Scott (R-Fla.
Drugs
BridgeBio Pharma plans to file an NDA next year after reporting positive data from Phase 3 FORTIFY trial evaluating BBP-418, an oral small-molecule therapy for limb-girdle muscular dystrophy type 2I/R9. The progressive genetic muscular dystrophy is caused by FKRP mutations, which impair glycosylation of alpha-dystroglycan
Devices
FDA releases a draft guidance outlining recommendations for medical device manufacturers on the type of Quality Management System (QMS) information to include in premarket submissions. The agency says the document is intended to help companies prepare and maintain a QMS and provide sufficient information for FDA to assess compliance with
Drugs
FDA has posted a six-item Form FDA-483 after inspecting Hetero Labs’ Vishakapatnam, India, warehouse last month. The findings from the inspection are: * Access to the facility storing active pharmaceutical ingredients, intermediates and key starting materials intended for the U.S. market and associated records were not made readily
Drugs
FDA has approved an expanded indication for Merck’s pulmonary arterial hypertension (PAH) therapy Winrevair (sotatercept-csrk), clearing the drug to reduce the risk of major clinical worsening events, including PAH-related hospitalization, lung transplantation and death. Approval was based on data from the Phase 3 ZENITH trial, which enrolled
Devices
FDA has cleared a YorLabs 510(k) for its new intracardiac imaging system that the company says could reshape workflows in catheterization labs by eliminating the need for costly capital equipment. The platform, which YorLabs describes as the first “zero-CapEx” intracardiac echocardiography (ICE) solution, delivers real-time, high-resolution
Drugs
Inhibrx Biosciences is reporting favorable data for treating chondrosarcoma with its antibody therapy ozekibart (INBRX-109). New data from the Phase 3 ChonDRAgon trial showed the therapy met the primary endpoint in advanced or metastatic, unresectable disease. According to the topline data, treatment cut the risk of progression or death
Drugs
FDA has posted a two-item Form FDA-483 from an August inspection of Sumitomo Pharma’s Suzuka, Japan, manufacturing facility that cited GMP deficiencies. The agency cited the firm over its sampling procedures involving incoming raw materials and excipients. It said Sumitomo had not established a documented rationale or
Drugs
A payroll system error has delayed paychecks for some FDA employees who are designated to continue working during the ongoing government shutdown, according to reporting from Federal News Network. Shutdown-exempt staff at FDA are typically paid on time using carryover funds. However, HHS spokesperson Emily Hilliard told the media