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