Monday is likely to be a sad day. An experimental drug to treat some boys with Duchenne muscular dystrophy will be evaluated by a panel of experts convened by the Food and Drug Administration. Based on briefing documents made available by the FDA this morning, it looks very likely they will vote to reject the drug – and that the FDA will follow that recommendation. Sarepta shares are down 42%.
If that rejection happens, the hopes of patients and their families, who have campaigned tirelessly for the approval of the medicine, which is called eteplirsen, will be dashed. It didn’t have to be this way. The handling of this situation reflects badly on the drug’s maker, Sarepta Therapeutics, and on the FDA. It points to some very fixable flaws in our drug regulation system, many of them involving the way that the FDA communicates with the public.
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