FDA advisers narrowly expedite approval of Sarepta gene therapy -Dlight News

FDA advisers narrowly expedite approval of Sarepta gene therapy

Advisers to the US Food and Drug Administration voted 8-6 on Friday to recommend accelerated approval of Sarepta Therapeutics Inc.’s gene therapy for Duchenne muscular dystrophy despite questions about its clinical benefit and safety. If approved, the potentially first-of-its-kind one-time experimental therapy, called SRP-9001, could radically change how patients with DMD are treated. Earlier this week, the FDA said Sarepta did not offer “unequivocal evidence” the therapy would benefit patients with the muscle-wasting malignancy. Sarepta SRPT, -0.88% , which hopes to receive fast-track approval by the FDA based on limited current data, is conducting late-stage trials to confirm the therapy’s benefits in patients. The first batch of data from the trial is expected by December; Full results should be available in early 2024. The FDA is expected to make a decision by May 29. The agency generally reflects the advice of its advisors, although it is not bound to do so. “Today’s advisory committee outcome is extremely important to the patient community, which urgently needs new therapies,” Sarepta Chief Executive Doug Ingram said in a statement late Friday. “With a May 29 action date as our top priority, we will work collaboratively with the FDA to complete the review of our BLA for SRP 9001.” Shares of Sarepta were flat in extended trading on Friday and are down 7% so far this year.

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