The unilateral decision by Peter Marks, a top official at the Food and Drug Administration, to approve the gene therapy Elevidys, a treatment for Duchenne muscular dystrophy, despite the misgivings of his staff, looks even more surprising on a close examination.
Marks’ decision to overrule three different review teams appears unprecedented in recent FDA history. But the agency declined to answer questions about why a process for appealing Marks’ decision was not invoked despite the disagreements. The agency also did not explain why it did not invoke the opinions of a committee of outside advisers, something that it often does in this kind of situation. Nor did the FDA answer questions from STAT on what precedents exist for Marks taking such dramatic action. (The FDA made a memo written by Marks about the approval public, as well as the three memos opposing approval, as a result of the FDA’s normal processes.)
The answers to these questions may not have any effect on Sarepta Therapeutics, Elevidys’ maker, which has seen its stock price increase 36% since the decision was announced on June 20. Approvals, once granted, are rarely revoked. But experts worry about the effect the decision will have on future FDA reviews, on the agency’s standards, and on the public’s trust.
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