MedCity News June 21, 2024
FDA reviewers concluded the data for Sarepta Therapeutics’ Elevidys were insufficient to show efficacy in the rare muscle disorder Duchenne muscular dystrophy. Peter Marks, the agency’s top biologics official, reviewed the same data and reached a different conclusion.
A Sarepta Therapeutics gene therapy that failed its confirmatory test has now converted its accelerated FDA approval into a traditional one, expanding use of the treatment to a wider group of Duchenne muscular dystrophy patients. In approving the therapy, the FDA’s top biologics official cited a wider range of supporting evidence, overruling agency staff who were unconvinced that the data show the therapy works to treat the muscle-wasting disease.
The FDA decision announced late Thursday permits use of the therapy, Elevidys, in...
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