The U.S. Food and Drug Administration cleared a shortened version of a gene for a muscle protein to be used in 4- and 5-year-olds with muscular dystrophy.
The $3.2 million one-time treatment is the first gene therapy approved to treat DMD, a progressive and fatal condition that manifests in early childhood.
By Leroy Leo and Aditya Samal (Reuters) -The U.S. Food and Drug Administration on Thursday granted accelerated approval to Sarepta Therapeutics' first.
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.