FDA advisers narrowly back accelerated approval of Sarepta gene therapy

U.S. Food and Drug Administration advisers voted 8-6 Friday to recommend the agency grant accelerated approval to Sarepta Therapeutics Inc.’s gene therapy for Duchenne muscular dystrophy despite questions about its clinical benefit and safety.

Should it be approved, the first-of-its-kind potential 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 “unambiguous evidence” the therapy will benefit patients with the deadly muscle-wasting disease.

which hopes to gain fast-track approval via the FDA based on limited current data, is conducting a late-stage trial to confirm the therapy’s benefits to 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 usually mirrors the  advice of its advisers, though it is not obligated.

“Today’s advisory committee outcome is extremely important to the patient community, who are in urgent need of new therapies,” said Sarepta Chief Executive Doug Ingram said in a statement late Friday. “With the May 29 action date 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 Friday, and are down 7% so far this year.

Read more from source