FDA Approves Sarepta’s Gene Therapy for Rare Muscular Dystrophy in Some Kids

The U.S. Food and Drug Administration in White Oak, Md., on July 20, 2020. Sarah Silbiger/Getty Images

The U.S. health regulator has granted accelerated approval to Sarepta Therapeutics’ first-of-its-kind gene therapy for Duchenne muscular dystrophy (DMD), an inherited progressive muscle-wasting disorder that almost always affects young boys.

Sarepta said on Thursday the Food and Drug Administration had approved the treatment for children aged between 4 and 5 years who can walk. It was initially seeking approval for all DMD patients who can walk.