FDA's Peter Marks overrode reviewers’ call to reject Sarepta’s new Duchenne gene therapy
Peter Marks, the senior FDA official who oversees the regulation of gene therapies, overrode agency reviewers’ decision to reject Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy after he disagreed with parts of their interpretation of the limited efficacy data.
The FDA on Thursday granted accelerated approval to Sarepta’s gene therapy, to be marketed as Elevidys, in boys aged 4 and 5 years old. The decision followed months of regulatory twists and a close vote by an advisory committee.
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