Up­dat­ed: FDA re­port rais­es con­cerns over Sarep­ta Duchenne gene ther­a­py ahead of ac­cel­er­at­ed ap­proval de­ci­sion

Ahead of what’s cer­tain to be a close­ly watched ad­vi­so­ry com­mit­tee meet­ing Fri­day, the FDA out­lined con­cerns on the ef­fi­ca­cy and safe­ty of Sarep­ta Ther­a­peu­tics’ gene ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy.

In brief­ing doc­u­ments re­leased Wednes­day, the FDA said there was a lack of “un­am­bigu­ous ev­i­dence” that Sarep­ta’s gene ther­a­py, known as SRP-9001, is like­ly ben­e­fi­cial for pa­tients with Duchenne. The FDA raised myr­i­ad con­cerns over whether clin­i­cal tri­al re­sults trans­lat­ed in­to ben­e­fit for pa­tients, along­side safe­ty and man­u­fac­tur­ing is­sues re­lat­ed to the vi­ral vec­tors used to de­liv­er the treat­ment.

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