FDA changes course on Sarepta’s once-rejected muscular dystrophy drug
The Food and Drug Administration reversed its decision on a treatment for Duchenne muscular dystrophy from Sarepta Therapeutics, approving a previously rejected drug without explaining what the problem was in the first place.
The therapy, called Vyondys 53, is approved to treat the roughly 8% of Duchenne patients whose disease results from a specific DNA error. In a clinical trial, Sarepta’s drug produced a small increase in an important muscle protein called dystrophin that is normally missing in children with Duchenne. The company has yet to demonstrate that Vyondys 53 can improve muscle function or slow the progression of the disease.
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