Drugs
FDA has issued a clinical hold against Athenex and Baylor College of Medicine’s kUR-501, an autologous GD2 CAR-NKT cell therapy for relapsed/refractory high-risk neuroblastoma (R/R HRNB), after a young heavily pretreated male patient died. The death came after treatment at the fifth dose level
Devices
Device manufacturers should use FDA’s remote regulatory assessment (RRA) program as a template for how the agency will likely implement remote device facility inspection authority under last year’s Food and Drug Omnibus Reform Act (FDORA), according to former CDRH Office of Compliance director Steven Silverman. Writing in a
Drugs
FDA reviewers appear optimistic that Biogen’s amyotrophic lateral sclerosis (ALS) drug tofersen could be considered for accelerated approval despite it failing to show a statistically significant difference from placebo groups for the primary and secondary endpoints in a recent study in patients with the superoxide dismutase 1 (SOD1) gene,
Biologics
FDA has told Sarepta Therapeutics that it has decided to hold an advisory committee meeting to review its gene therapy BLA for SRP-9001 (delandistrogene moxeparvovec), indicated for treating Duchenne muscular dystrophy. Previously when the submission was accepted for review, the agency said that an advisory committee meeting was not
Devices
FDA has issued Olympus Medical Systems a Warning Letter after inspecting the firm’s Tokyo facility and documenting GMP deficiencies. According to the 3/15 letter, the 11/2022 inspection cited Olympus for failing to adequately establish and maintain procedures for implementing corrective and preventive actions. It also failed to
Biologics
An Eastern PA federal judge has approved a $25 million class action settlement involving Johnson & Johnson’s Remicade (infliximab). The lawsuit (In re Remicade Antitrust Litigation, No. 2:17-cv-04326-KSM) claimed that defendants Johnson & Johnson and Janssen Biotech violated federal and state antitrust and consumer-protection
Drugs
CDER has published its Office of Compliance (OC) Annual Report for fiscal year 2022. The report says OC relies on three strategic priorities to guide decision-making in support of its mission. The first priority is to promote compliance by proactively promoting compliance through communication with stakeholders, including through publishing
Devices
Datascope/Getinge has recalled its Cardiosave Hybrid IABPs and Rescue IABPs because the coiled cable connecting the display and base on some units may fail, causing an unexpected shutdown. “An unexpected pump shutdown and any interruption to therapy that occurs can lead to unstable blood flow (hemodynamic instability), organ damage
Drugs
FDA has accepted for review an Ipsen resubmitted NDA for palovarotene as a potential treatment for fibrodysplasia ossificans progressiva (FOP). The ultra-rare disease causes permanent and continuous bone formation in soft and connective tissues like muscles, tendons and ligaments. The agency has set a user fee review action date
Drugs
FDA has published a guidance, Frequently Asked Questions About Medical Foods, to give industry a convenient place to find answers to frequently asked questions about medical foods. The document is a third edition of a 5/2007 guidance with the same name. It addresses common questions about the definition of
Drugs
FDA has approved a Telix Pharmaceuticals supplemental NDA for Illuccix (kit for the preparation of gallium Ga 68 gozetotide injection) for use in selecting patients with metastatic prostate cancer for whom Novartis’ Pluvicto is indicated. To qualify to be treated with the prostate-specific membrane antigen (PSMA)-directed radioligand Pluvicto,
Devices
FDA has granted 510(k) clearance for the BD Vaginal Panel on the BD COR system, a comprehensive diagnostic test that is said to directly detect the three most common infectious causes of vaginitis using BD’s high-throughput molecular diagnostic platform for large laboratories. A company statement says the
Biologics
GlaxoSmithKline says its meningococcal ABCWY vaccine candidate met all 11 primary endpoints in a pivotal Phase 3 trial and was well tolerated with a safety profile consistent with the company’s Bexsero and Menveo meningococcal vaccines. A company statement says the primary endpoint data demonstrated non-inferiority compared to Bexsero
Biologics
FDA has published an International Pharmaceutical Regulators Program (IPRP) reflection paper, General Considerations for Raw Materials Used in the Manufacture of Human Cell and Gene Therapy Products, to provide the perspectives of the IPRP cell therapy working group and gene therapy working group on the quality of raw materials used
FDA Policy/General
The Senate Appropriations Committee has announced a 4/19 Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Subcommittee hearing to review FDA’s fiscal year 2024 budget request. While it is still too early for witnesses to be announced, typically FDA commissioner Robert Califf would be the primary
Drugs
FDA’s Antimicrobial Drugs Advisory Committee 3/16 voted 16 to 1 that Pfizer’s Paxlovid (nirmatrelvir and ritonavir) has a favorable benefit-risk profile for treating mild-to-moderate Covid-19 in adults who are at high risk for progression to severe disease. The company’s submission is seeking
Drugs
FDA commissioner Robert Califf says insurers and other stakeholders have a role to play in ensuring that studies on new drugs approved through the accelerated approval pathway are completed. Speaking 3/16 to the AHIP Medicare, Medicaid, Duals & Commercial Markets Forum, Califf said completion of the required studies is
Drugs
FDA has published a draft guidance, Development of Local Anesthetic Drug Products with Prolonged Duration of Effect, to assist sponsors that are developing local anesthetic drug products to produce postoperative analgesia for a prolonged duration, for which submission of a 505(b) NDA is appropriate. The document says its provisions
FDA Policy/General
FDA has published a draft guidance, Electronic Systems, Electronic Records, and Electronic Signatures in Clinical Investigations Questions and Answers, with guidance for sponsors, clinical investigators, institutional review boards, contract research organizations, and other interested parties on using electronic systems, records, and signatures in clinical investigations of medical products, food, tobacco
Drugs
FDA has modified a partial clinical hold against Stoke Therapeutics to allow the administration of a higher single dose of STK-001 (70 mg) in its ongoing Phase 1/2a MONARCH study of children and adolescents with Dravet syndrome. “This new 70 mg single dose cohort in MONARCH will complement
Drugs
Sens. Rand Paul (R-KY) and Maggie Hassan (D-NH) have reintroduced the Transparency in Generic Drug Applications Act that they say would help bring generic medications onto the market faster and lower prescription drug prices for consumers. In a statement (includes a link to the bill text), Paul and
Drugs
Former pharmaceutical sales representative Vincent Tornari of Linwood, NJ, pled guilty 3/14 to one count of an indictment charging him with conspiring to commit healthcare fraud and wire fraud. A Department of Justice statement says Tornari and other conspirators who were healthcare providers and wrote fraudulent prescriptions learned that