STAT+: How controversial was the decision by FDA’s Peter Marks to approve Sarepta’s gene therapy? Check its footnotes

STAT July 2, 2024
Matthew Herper

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...

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