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Slim Adcomm Majority Boosts Sarepta’s Gene Therapy In Duchenne Muscular Dystrophy

 
• By Derrick Gingery

Accelerated approval seems imminent after a majority of the US FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted the risk-benefit balance supported it.

Sarepta SRP-9001 AC vote
An FDA official announces the advisory committee vote. • Source: Screenshot of FDA broadcast

Despite significant uncertainty about the efficacy of Sarepta Therapeutics, Inc.’s proposed gene therapy in Duchenne muscular dystrophy, the product took a big step toward an accelerated approval thanks to a slim majority of advisory committee votes.

Members of the US Food and Drug Administration’s Cellular, Tissue and Gene Therapies Advisory Committee voted 8-6 that the

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