Last night, a panel of advisors to the Food and Drug Administration voted 7 to 3, with 3 abstentions, that the agency should not approve eteplirsen, a drug for a Duchenne muscular dystrophy, a rare, deadly, muscle-destroying disease, developed by Sarepta, a Cambridge, Mass.-based biotechnology firm. (On another vote, which could justify a conditional approval, the tally was closer: 7-6, still against.)
The vote was a recommendation, but the FDA will likely follow it. That would crush the hopes of many boys (Duchenne is a disease of boys) and their families. Patient advocates, including the moms of boys who received eteplirsen in clinical trials, have campaigned passionately that the drug should be approved.
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