Attorneys say Sarepta did not have an obligation to report its appeal, particularly since winning a formal dispute filing with the US FDA is a long shot.

Sarepta must perform enhanced pharmacovigilance for serious renal toxicity events and rhabdomyolysis with the Duchenne muscular dystrophy drug, which carries a label warning for renal toxicity. Approval letter includes 2024 target completion date for ESSENCE confirmatory trial.

Efficacy data were striking to committee members, but they want to ensure postmarketing plans gather adequate data to address safety questions.

The latest drug development news and highlights from our US FDA Performance Tracker.

Five new drugs have been recommended for EU approval at the latest meeting of European Medicines Agency’s drug evaluation committee.

Recent and upcoming US FDA advisory committee meetings and a summary of topics covered.

Revised North American trade deal touted as enabling Congress to address crisis of high drug prices; requirement for 10 years biologic exclusivity is removed and language to allow incentives for generics added.

In case that could impact challenges of biopharma patents, Supreme Court questioned the consequences of not allowing courts to review PTAB institution decisions when petition may be time barred.

The $9.7bn acquisition hinges on the small interfering RNA inclisiran, but an ongoing cardiovascular outcomes trial, manufacturing hurdles, and reimbursement challenges provide subjects for concern.

White House Council of Economic Advisors says US House drug price “negotiation” bill will reduce the number of innovative medicines by 100 over a decade; the Congressional Budget Office says more like 8-15. Both analyses miss the point – but also show that industry is losing a crucial piece of political ground.

The $9.7bn acquisition hinges on the small interfering RNA inclisiran, but an ongoing cardiovascular outcomes trial, manufacturing hurdles, and reimbursement challenges provide subjects for concern.

Other NASH registration trails have targeted fibrosis, but Galectin's mostly-resolved discussions with US FDA will allow the belapectin program to focus of signs and symptoms of the disease.

Sanofi spends 70% of vaccine production on quality control, Sanofi Pasteur exec VP David Loew tells the Pink Sheet; company is working on next generation flu vaccines that may provide greater efficacy.

Rather than blame employees for ignoring 397 product complaints, Teligent should have fixed complaint handling system, US FDA warning letter says.

Health ministers have called for more action on access to medicines, including a new EU work agenda, rebuilding Europe’s manufacturing capabilities, further action on pricing, and greater leeway for member states to mitigate drug shortages by restricting parallel exports.

US FDA officials tell industry that implementing a quality metrics program is an agency priority and that a rejiggered program will incorporate various elements of other quality metrics initiatives being piloted by the agency, industry and academia.