All articles tagged with #srp 9001
Sarepta Therapeutics is awaiting FDA approval for its gene therapy for Duchenne muscular dystrophy, with the decision expected to be announced today. The approval is likely to be limited to boys aged 4 and 5 years old, rather than the broader subset of patients originally studied in trials. The therapy, SRP-9001, is a one-time gene therapy for young boys with the debilitating muscle disease.
Sarepta Therapeutics' shares jumped 29% in pre-market trading after an FDA advisory committee backed the company's marketing application for gene therapy SRP-9001, which is targeted at certain patients with Duchenne muscular dystrophy. The FDA is due to make a final decision on the treatment before May 29, 2023. Sarepta's manufacturing partner, Catalent, also saw a rise in its stock price. However, concerns remain that a potential failure of the company's ongoing Phase 3 trial for SRP-9001 could force Sarepta to pull the treatment from the market.