Sarepta Therapeutics' Therapy Becomes First To Score FDA Nod For Duchenne Muscular Dystrophy Gene Therapy
Portfolio Pulse from Vandana Singh
The FDA has granted accelerated approval to Sarepta Therapeutics' gene therapy, Elevidys, for Duchenne muscular dystrophy (DMD) in pediatric patients aged 4-5 years with a confirmed mutation in the DMD gene. Continued approval depends on further verification and description of clinical benefit in confirmatory trials. Sarepta is preparing to launch Elevidys and plans to submit a BLA supplement to expand the approved label based on the results of the confirmatory trial.
June 22, 2023 | 6:48 pm
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Sarepta Therapeutics' Elevidys receives FDA accelerated approval for Duchenne muscular dystrophy in pediatric patients. Continued approval depends on confirmatory trials.
The FDA's accelerated approval of Elevidys is a significant milestone for Sarepta Therapeutics, as it becomes the first gene therapy for Duchenne muscular dystrophy. This approval could lead to increased demand for the therapy and potential revenue growth for the company. However, continued approval depends on the results of confirmatory trials, which adds some uncertainty to the long-term prospects of the therapy.
CONFIDENCE 90
IMPORTANCE 80
RELEVANCE 100