Federal health advisers have sided against an experimental treatment for muscular dystrophy, the latest in a series of setbacks for the Sarepta Therapeutics’ drug.
The panel of experts to the Food and Drug Administration voted 7-3 Monday that the drug was not shown to be effective for the debilitating degenerative disease. Three panelists abstained from voting.
While not binding, the panel’s view carries weight in FDA’s final decision whether to approve a drug, which has become a rallying point for patients and families affected by the deadly disease
The vote followed a decidedly negative FDA review posted online last week. It concluded that “there does not appear to be any evidence of efficacy” for the drug.