Internal documents shed new light on Sarepta FDA decision, which top agency official says was ‘exceptional’
While he has repeatedly advocated for accelerated gene therapy approvals, the FDA’s top cell and gene therapy official Peter Marks said that the agency’s landmark decision to approve Sarepta’s Duchenne gene therapy shouldn’t be viewed as an example of a usual accelerated approval.
“I think that was exceptional. As you may be aware of from all the memos that are now public, there was a lot that went around that approval. I think we’ll use age restrictions only when it absolutely makes sense from a safety perspective and an efficacy perspective,” Marks, the director of the FDA’s Center for Biologics Evaluation and Research (CBER), said at Biopharma Congress on Monday.
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