Drugs
FDA has accepted for review a Capricor Therapeutics BLA resubmission for its experimental cell therapy deramiocel, intended to treat heart complications in patients with Duchenne muscular dystrophy. FDA classified the filing as a Class 2 resubmission and set a new user fee review action target date of 8/22. The
Drugs
FDA has approved an expanded use of GSK’s Wellcovorin (leucovorin calcium) tablets for treating cerebral folate deficiency in adult and pediatric patients who have a confirmed variant in the folate receptor 1 gene (CFD-FOLR1). The approval fell short of agency and HHS expectations communicated in September that hinted
Drugs
A 9/18-9/26 FDA inspection at the Simtra BioPharma Solutions drug manufacturing facility in Westphalia, Germany, found significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A 3/3 Warning Letter says the specific violations were: * failure to perform operations within specifically defined areas of
Drugs
A 1/13-2/7/2025 FDA inspection at the Novo Nordisk facility in Plainsboro, NJ, identified serious violations of postmarketing adverse drug experience (PADE) reporting requirements. A 3/5 Warning Letter cites several examples of how Novo Nordisk failed to develop written procedures for the surveillance, receipt, evaluation, and
Drugs
An 8/4-8/12/2025 FDA inspection at the Fareva drug manufacturing facility in Morton Grove, IL, identified significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A 3/3 Warning Letter says the three specific violations were: * failure to use equipment in the manufacture, processing,
Devices
FDA has issued an early alert regarding a potential safety issue with certain Erbe USA Flexible Cryoprobes, following reports that some devices may rupture during use. FDA says Erbe USA has advised customers to remove specific Flexible Cryoprobes from use or sale. The devices are used to destroy tissue through
FDA Policy/General
The Institute for Safe Medicines says FDA, the Federal Trade Commission, and the Department of Justice should take immediate action against Hims & Hers for its Super Bowl ad that promoted unapproved compounded drug products. A 3/3 letter to FDA says the ad “contained a startling array of unsubstantiated
FDA Policy/General
Five Sidley attorneys say the FDA 9/2025 crackdown on direct-to-consumer (DTC) prescription drug ads has involved Warning and Untitled Letters that were issued to drug manufacturers that articulate positions that “raise significant constitutional, legal, and regulatory concerns.” Their legal update notes that a recent Untitled Letter directly
Biologics
FDA has released an updated draft guidance outlining new and revised recommendations for companies developing biosimilar products, including clarifications on the use of foreign comparator products in clinical studies and requirements for retaining study samples. The document revises and replaces a 2021 draft guidance titled New and Revised Draft Q&
Biologics
FDA has issued Incyte a complete response letter (CRL) for its supplemental BLA for Zynyz (retifanlimab-dlwr) injection (375mg) for an additional indication for treating adult patients with metastatic non-small cell lung cancer in combination with platinum-based chemotherapy. The sBLA was based on data from the Phase 3
Drugs
Consumer advocacy group Public Citizen is pressing Pfizer to commit to maintaining access to its medicines in Europe after comments by CEO Albert Bourla suggesting the company could stop supplying drugs to France if governments refuse to pay higher prices. In a letter sent Monday, the group demanded that Pfizer
Drugs
Roche is reporting that its investigational breast cancer drug giredestrant failed to significantly extend progression-free survival in a Phase 3 study when combined with the CDK4/6 inhibitor palbociclib (Pfizer’s Ibrance), dealing a setback to the company’s effort to establish the therapy in first-line metastatic disease.
Drugs
FDA says it recently held a meeting with several states to discuss implementation of a program that would allow the importation of certain lower-cost prescription drugs from Canada. The discussions focused on the Section 804 Importation Program, which permits states and Native American tribes to seek authorization to import
Drugs
Xenon Pharmaceuticals says its experimental epilepsy drug azetukalner met the primary endpoint in a late-stage clinical trial in patients with focal onset seizures, paving the way for a planned U.S. regulatory filing later this year. Topline results from the Phase 3 X-TOLE2 study showed the drug significantly
Drugs
Ipsen is voluntarily withdrawing Tazverik (tazemetostat) from the market based on emerging data from the ongoing Phase 1b/3 SYMPHONY-1 trial that is evaluating the therapy in combination with lenalidomide plus rituximab in follicular lymphoma. The trial’s independent data monitoring committee advised that based on adverse events of
Drugs
FDA investigators have cited manufacturing and documentation issues at an Indian sterile drug facility operated by Maiva Pharma Private Ltd. following an inspection last month, according to an inspection report issued by the agency. The inspection, conducted from 2/10-20 at the company’s plant in Hosur in the
FDA Policy/General
Americans express significantly greater confidence in career scientists at federal health agencies than in the leaders who oversee them, according to a new national survey that also shows declining trust in the FDA and other federal health regulators over the past two years. The survey, released 3/5 by the
Biologics
CBER director Vinay Prasad will step down at the end of April, according to HHS. His departure comes on the heels of several recent controversial decisions at the Center and news of an internal investigation into complaints from staff. HHS did not provide additional details about the reason for his
Drugs
An FDA official discussing an ongoing dispute this week with UniQure over its gene therapy for Huntington’s disease (see story) also raised concerns with outside experts who sit on agency advisory committees. The official, who spoke on condition of anonymity, criticized the advisory committees, suggesting some outside experts “do
Devices
FDA says an expanded recall by Medline Industries involving certain reprocessed electrophysiology and ultrasound catheters has been classified as Class 1, warning that the devices could cause serious injury or death if used. The recall affects several catheters distributed through Medline’s ReNewal reprocessing program, including models originally manufactured by
FDA Policy/General
FDA has issued a draft guidance outlining how drug manufacturers should respond to inspection findings documented in an FDA Form 483, urging companies to submit detailed remediation plans quickly and take a more systematic approach to correcting quality problems. The document describes best practices for responding to inspectional observations identified
FDA Policy/General
After a year at the agency, FDA deputy commissioner for operations and chief operating officer Barclay Butler has announced his retirement in the coming months, according to an internal email. FDA commissioner Marty Makary has named Butler’s deputy, Melanie Keller, to assume his duties effective 4/6. Before joining