In a May 13, 2014, webcast, Sarepta Therapeutics summarized its plans for eteplirsen and other experimental exon-skipping compounds it has in development to treat Duchenne muscular dystrophy; eteplirsen targets exon 51 of the dystrophin gene and could provide a treatment for 13% of the DMD population. Sarepta will apply to the U.S. Food and Drug Administration for accelerated approval of eteplirsen, which could be granted as early as 2015 but will require a confirmatory study for full approval; an accelerated approval designation allows patients to have access to the drug prior to full approval. By early 2015, Sarepta hopes to open trials of its compounds targeting dystrophin.
