Drugs
An 8/29-9/2/22 FDA inspection at the drug manufacturing facility maintained by Dunagin Pharmaceuticals, doing business as Massco Dental, in Rogers, AR, found significant violations of current good manufacturing practice (CGMP) regulations for finished pharmaceuticals. A just-released 2/22 Warning Letter says specific violations were: * failing
Drugs
A 9/6-9/20/22 FDA inspection at the Cosmetic Science Laboratories drug manufacturing facility in Torrance, CA, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A 3/10 Warning Letter says specific violations were: * failing to conduct at least one test to verify
Biologics
Speaking on 3/20 to the 2023 Muscular Dystrophy Association Clinical and Scientific Conference, CBER director Peter Marks discussed three ways FDA is working to overcome barriers to developing gene therapies for rare diseases. In a video summary of his keynote address posted by Neurology Live, Marks said FDA is
Drugs
CDER is making several requests if FDA chief scientist Namandjé Bumpus grants an Intarcia Therapeutics 2/21 petition seeking a public hearing before the FDA Endocrinologic and Metabolic Drugs Advisory Committee on the Center’s refusal to approve an Intarcia NDA for its ITCA 650 (exenatide in DUROS device). Intarcia
Drugs
Karuna Therapeutics is planning a mid-year NDA for schizophrenia drug KarXT (xanomeline-trospium) after reporting positive topline results from its Phase 3 EMERGENT-3 trial evaluating the drug's (XXXX INSERT XXX)efficacy, safety (XXX DELETE COMMA XXXX)and tolerability. (XXXX DELETE XXX) “The trial met its primary
Drugs
Pharmaceutical Research and Manufacturers of America (PhRMA) says rare disease drug development could benefit by FDA providing greater detail and context on the types of evidence that may be considered confirmatory. In comments on rare disease development, the industry group says that because many rare diseases do not have relevant
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
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,
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
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,
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
Drugs
FDA reviewers say NDA data support the efficacy of Pfizer’s Paxlovid (nirmatrelvir and ritonavir) for treating mild-to-moderate Covid-19 in adults who are at high risk for progression to severe disease. The company’s data also demonstrated that Paxlovid retained antiviral activity against the Omicron variant and
Drugs
As required by the Orange Book Transparency Act of 2020, the Government Accountability Office (GAO) has reviewed FDA Orange Book patent listings for drug/device combination products, including the listings’ implications for generic products. In a report, GAO says it interviewed 15 stakeholders on (1) how Orange Book patent listings
Drugs
Pharmaceutical Research and Manufacturers of America (PhRMA) says a revised FDA draft guidance on the content and format of the Dosage and Administration section of labeling for human prescription drug and biological products provides more detail and direction than its previous 2010 document. PhRMA says the revised guidance “will provide