By Gina Shaw
Overruling the recommendation of its advisory panel, the FDA on Sept. 19 granted approval to eteplirsen (Exondys 51, Sarepta), the first drug to directly treat Duchenne muscular dystrophy, the most severe and lethal form of the degenerative neuromuscular disorder.
The approval, which came four months after the drug’s planned May Prescription Drug User Fee Act date was delayed, notes that the drug is indicated for the approximately 13% of the Duchenne population who have