By Steve Usdin, Washington Editor and Susan Schaeffer, Editor, BioCentury
The skimpy NDA for eteplirsen to treat Duchenne muscular dystrophy has FDA wedged between the rock of inconclusive data and the hard place of a well-informed patient community that understands the limitations of the data and still demands access to the compound. There are very good arguments for rejecting eteplirsen, but they are outweighed by the possibility that the compound might help and is very unlikely to harm boys who have no other hope.
In the interest of patients, FDA should grant accelerated approval to eteplirsen for DMD that is amenable to exon 51 skipping. The agency should couple the approval with stringent requirements for Sarepta Therapeutics Inc. to rigorously confirm clinical efficacy, as well as an unambiguous understanding that approval will be withdrawn if efficacy is not confirmed.
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- N = 1