Posted on: 13 October 2017, source: ScienceMag
A pioneering AAV gene therapy from Spark Therapeutics took a giant stride toward an FDA approval yesterday as an outside panel of experts offered their support for getting this game-changing treatment into the market after looking over the data and hearing from some of the severely sight-impaired patients whose lives had been transformed by this therapy. The vote was 16 to 0 favoring the benefit-risk profile of the drug, backing an OK for voretigene neparvovec by the agency’s Cellular, Tissue and Gene Therapies Advisory Committee and providing a compelling reason for the U.S. Food and Drug Administration to follow through with an historic first U.S. approval of a vector-delivered gene therapy.