Drugs
Writing in an FDA Voices column, four senior agency executives describe ways in which FDA is strengthening its oversight of good clinical practice (GCP) as the ethical and scientific foundation for clinical investigations of all regulated products. The column was written by CDER acting director Michael Davis, CBER acting director
Drugs
The American Society of Gene & Cell Therapy (ASOCT) says an FDA draft guidance on leveraging prior knowledge in developing human gene therapy products incorporating genome editing would have greater value if it applied more broadly across cell and gene therapy modalities and provided greater clarity about how knowledge accumulated
Drugs
CBER has published a Standard Operating Policy and Procedure (SOPP), Complying with Requirements Under the Pediatric Research Equity Act (PREA) & Best Pharmaceuticals for Children Act (BPCA), as a resource to ensure that sponsors/applicants and CBER staff follow the required procedures and timeframes under the two laws. The SOPP
Drugs
A Friends of Cancer Research (Friends) white paper proposes a fit-for-purpose approach to support administered activity escalation and dosage optimization in radiotherapeutic therapy (RPT) development. Friends says its approach is based on these core principles: * use escalation to generate RPT-specific evidence; * anchor decisions to the totality of
Drugs
CDRH has issued an alert on AVID Medical convenience kits based on a letter the company sent to customers recommending that certain kits be corrected prior to continued use by removing components affected by the earlier Huons Co., Ltd. recall. The Center says users should not use sodium chloride ampules
Biologics
uniQure has submitted a BLA seeking accelerated approval of ifezuntirgene inilparvovec (AMT-130), an investigational gene therapy for treating Huntington’s disease. The company’s submission requested a priority review. The filing is based on a three-year data analysis from a Phase I/II clinical study of ifezuntirgene inilparvovec,
Drugs
FDA has ordered a Boxed Warning for American Regent’s Injectafer (ferric carboxymaltose injection) over the risk of symptomatic hypophosphatemia, a potentially serious condition in which phosphate levels in the blood become abnormally low. The warning recommends monitoring blood phosphate levels in patients considered at risk for the condition, as
enforcement
A 4/13-4/17 FDA inspection at the Shoolin Pharma Chem drug manufacturing facility in Mehsana, Gujarat, India, found that the company manufactures active pharmaceutical ingredients (APIs) under insanitary conditions and with significant deviations from current good manufacturing practice (CGMP) regulations for APIs. An 8/18 Warning Letter says
Drugs
FDA has released a draft guidance entitled Pharmacokinetics in Patients with Impaired Hepatic Function: Study Design, Data Analysis, and Impact on Dosage. The document is intended to assist sponsors in their design and analysis of studies that assess the influence of impaired hepatic function (hepatic impairment (HI)) on the pharmacokinetics
Devices
FDA has released two guidance documents on dental-related medical devices. The one guidance, Dental Curing Lights – Premarket Notification (510(k)) Submissions, provides recommendations for 510(k) submissions for dental curing lights that emit non-ionizing optical radiation intended to photopolymerize dental restorative resins. FDA is issuing this guidance to
Drugs
A 3/2-3/6 FDA inspection at the Fresenius Medical Care North America drug manufacturing facility in Ogden, UT, identified significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. An 8/25 Warning Letter says the two specific violations were: * failure to thoroughly investigate any unexplained
Devices
FDA has added a new frequently asked questions section to its Web page on PFAS (per- and polyfluoroalkyl substances) in medical devices, saying it has found no evidence that certain PFAS materials widely used in medical equipment are harming patients. The agency said its review of scientific literature and post-
Drugs
Novartis and Bristol Myers Squibb have each paused some studies of CAR T candidates while adverse events are evaluated. BioSpace reports Novartis suspended multiple studies of its rapcabtagene autoleucel (rap-cel) after three patients who received the therapy suffered fatal cases of immune effector cell-associated hemophagocytic syndrome. Meanwhile, the
enforcement
A 2/23-3/6 FDA inspection at Jabil Inc.’s Pharmaceutics International drug manufacturing facility in Cockeysville, MD, found significant violations of current good practice (CGMP) regulations for finished drugs. An 8/27 Warning Letter says the three specific violations were: * failure to thoroughly investigate any unexplained discrepancy or
Drugs
A 2/19-2/27 FDA inspection at the Reliance Life Sciences Private Limited drug manufacturing facility in Navi Mumbai, Maharashtra, India, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. An 8/18 Warning Letter says the specific violations were: * failure to ensure that laboratory
Drugs
Clear rules are needed for when FDA decides to publicly release complete response letters (CRL), according to a new analysis published in the Journal of Law and the Biosciences. The CRL postings, which were launched under former FDA commissioner Marty Makary as part of a broader “radical transparency” effort, are
Drugs
FDA and the International Coalition of Medicines Regulatory Authorities (ICMRA) have completed an assessment of a new biologics testing approach that could reduce use of horseshoe crab blood while continuing to ensure product quality. The assessment is part of the ICMRA Collaborative Assessment Pilot, which brings participating international regulators together
Drugs
The Washington, DC Court of Appeals appears to have raised the bar for generic drugmakers seeking to preserve 180-day marketing exclusivity after missing its FDA-mandated approval deadline. In Norwich Pharmaceuticals Inc. v. Kennedy, the court ruled that a first generic applicant can avoid forfeiting its exclusivity for missing
FDA Policy/General
FDA and the U.S. Securities and Exchange Commission have agreed to strengthen cooperation and share information involving companies and products regulated by the agency. Under a new three-year memorandum of understanding, the agencies will establish procedures for exchanging both public and certain non-public information when it is
Drugs
Karyopharm Therapeutics has submitted a supplemental NDA seeking accelerated approval for Xpovio (selinexor) in combination with ruxolitinib for patients with myelofibrosis. Karyopharm has requested a priority review, which, if granted, could result in a six-month review. The sNDA is based, in part, on data from the Phase 3 SENTRY
Drugs
IDEAYA Biosciences says it had a successful FDA Type C meeting to determine the Phase 3 registrational trial design for IDE849, a delta-like ligand 3 (DLL3)-targeting Topo-I-payload antibody drug conjugate (ADC) and its use in extensive stage small cell lung cancer (ES-SCLC). IDEAYA is planning
Drugs
FDA has approved Priovant Therapeutics’ Lisraya (brepocitinib) tablets for treating dermatomyositis in adults. “For too long, patients with dermatomyositis have faced a significant unmet need for effective treatments, often relying on therapies meant for other diseases,” FDA says in a release. “Dermatomyositis is a rare autoimmune disease in which the