FDA ad­comm backs ac­cel­er­at­ed ap­proval of Sarep­ta's Duchenne gene ther­a­py in close vote

An FDA ad­vi­so­ry com­mit­tee vot­ed 8-6 on Fri­day that the ben­e­fits of Sarep­ta Ther­a­peu­tics’ gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy out­weigh its risks, lend­ing their back­ing to ac­cel­er­at­ed ap­proval of the treat­ment.

In a day-long meet­ing, out­side ex­perts heard ar­gu­ments over whether the FDA should al­low use of Sarep­ta’s gene ther­a­py, known as SRP-9001, based on a sur­ro­gate end­point for pa­tients with Duchenne mus­cu­lar dy­s­tro­phy who are able to walk and have a con­firmed mu­ta­tion.

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