STAT+: FDA staff leaned toward rejecting Sarepta gene therapy before top official intervened
Reviewers at the FDA were leaning toward rejecting a closely watched gene therapy for Duchenne muscular dystrophy made by Sarepta Therapeutics, prompting a top official to intervene earlier this year,…
Reviewers at the Food and Drug Administration were leaning toward rejecting a closely watched gene therapy for Duchenne muscular dystrophy made by Sarepta Therapeutics, prompting a top official to intervene earlier this year, according to three people with direct knowledge of the agency deliberations.
Inside the FDA office that oversees gene therapies, some staff had reached a non-binding conclusion that Sarepta’s gene therapy should be rejected, the individuals said, speaking on condition of anonymity. Peter Marks, a top FDA official and vocal advocate for faster gene therapy approvals, stepped in and directed staff to schedule a public hearing on the therapy on May 12.
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