ARTICLE | Regulation

Sarepta’s DMD saga casts more clouds on FDA’s decision-making 

 A slimmer label for Sarepta’s gene therapy could have big consequences

May 25, 2023 12:27 AM UTC

It’s hard to reconcile FDA’s decision to extend the review of Sarepta’s gene therapy for DMD, apparently to consider restricting the label to younger patients, with the data presented by the company. Such a move would not only add to the already shaky argument for accelerated approval, but is almost certain to propagate ongoing concerns about the scientific integrity of FDA’s accelerated approval process.

The change will get mixed reviews from caregivers. Some will surely embrace the development given FDA’s apparent leaning towards declining approval during the advisory committee meeting. But the decision would also have serious negative consequences for other Duchenne muscular dystrophy patients  — squashing hope of access for the patients who arguably need the therapy most urgently...

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Sarepta Therapeutics Inc.