FDA adcomm backs accelerated approval of Sarepta's Duchenne gene therapy in close vote
An FDA advisory committee voted 8-6 on Friday that the benefits of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy outweigh its risks, lending their backing to accelerated approval of the treatment.
In a day-long meeting, outside experts heard arguments over whether the FDA should allow use of Sarepta’s gene therapy, known as SRP-9001, based on a surrogate endpoint for patients with Duchenne muscular dystrophy who are able to walk and have a confirmed mutation.
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