Eteplirsen success in Phase III for Duchenne Muscular Dystrophy

Sarepta Therapeutics, Inc. has announced the first peer-reviewed publication of the 48-week results from the Phase IIb clinical study of eteplirsen in the Annals of Neurology.

Eteplirsen is an investigational medicine in development for the treatment of patients with Duchenne Muscular Dystrophy (DMD) who have a genotype amenable to skipping of exon 51. Published study results showed that once-weekly treatment with eteplirsen resulted in a statistically significant increase from baseline in novel dystrophin, the protein that is lacking in patients with DMD. In addition, eteplirsen-treated patients evaluable on the 6-minute walk test (6MWT) demonstrated stabilization in walking ability compared to a placebo/delayed-treatment cohort. Eteplirsen was well tolerated in the study with no clinically significant treatment-related adverse events. These data will form the basis of a New Drug Application (NDA) to the FDA for eteplirsen planned for the first half of 2014.