A federal advisory panel voted Monday that a drug from Sarepta Therapeutics was not effective for treating Duchenne muscular dystrophy, a rare and fatal muscle-wasting disease. About 13,000 children, mostly boys, are afflicted.
The vote came after a daylong session punctuated by emotional pleas from dozens of parents and their children, some of whom appeared in wheelchairs, to describe how the Sarepta drug, called eteplirsen, made a substantial difference in clinical trials. Their testimony was balanced by presentations from US Food and Drug Administration staff who took a dim view of the Sarepta trial data.
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