The Food and Drug Administration is convening a meeting of outside experts on Friday to review clinical data on an experimental gene therapy for Duchenne muscular dystrophy from Sarepta Therapeutics. The stakes for this all-day meeting are high and deliberations could be contentious.
The advisory panel meeting is one of the most pivotal moments in recent memory for Duchenne patients and their families, many of whom view Sarepta’s gene therapy as the best hope for slowing the fatal muscle-wasting disease. Some them will participate in the hearing.
It’s also controversial. Sarepta is the first company to ask the FDA to give a gene therapy accelerated approval, based on preliminary evidence. In documents released Wednesday, FDA reviewers concluded Sarepta’s clinical trials “do not provide unambiguous evidence” that the therapy, known as SRP-9001, is “likely beneficial for ambulatory patients” with Duchenne. The agency also raised some safety concerns.
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