FDA approves Sarepta’s Duchenne gene therapy for nearly all patients – STAT

The Food and Drug Administration on Thursday approved Elevidys, Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy, for nearly all patients with the fatal muscle-wasting disease, dramatically expanding the initial, narrow authorization handed down last year.

The decision will likely be greeted by many families and doctors as a turning point in the fight against the muscle-wasting disease, even if its not nearly the seismic shift some had hoped for just a couple of years ago.

Gene therapy had long been seen as one of the approaches that might cure or at least halt the fatal condition. But clinical trials have presented a far murkier reality. Three different randomized studies of Duchenne gene therapies, including the only two conducted with Elevidys, have failed to reach their primary endpoints. And reports have circulated of boys Duchenne mostly affects boys whose conditions begin to decline just a couple years after treatment.

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FDA approves Sarepta's Duchenne gene therapy for nearly all patients - STAT