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.
Sarepta announced the scheduling of the public hearing last month, but the internal deliberations that led to it have not been previously reported.
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