Sarepta Therapeutics To Submit NDA For Eteplirsen By Year End 2014 - Quick Facts

(RTTNews) - Sarepta Therapeutics, Inc. (SRPT), a developer of innovative RNA-based therapeutics, plans to submit a New Drug Application or NDA to the U.S. Food and Drug Administration or FDA by the end of 2014 for the approval of eteplirsen for the treatment of Duchenne muscular dystrophy or DMD.

Eteplirsen is Sarepta's lead exon-skipping drug candidate in development for the treatment of patients with DMD who have a genotype amenable to skipping of exon 51.

The plan to submit an NDA for eteplirsen by 2014 end is based upon a guidance letter from the Agency that proposed a strategy regarding the submission of an NDA for eteplirsen under a potential Accelerated Approval pathway and served as the final meeting minutes for four meetings that took place between November, 2013 and March, 2014. The Agency noted that "with additional data to support the efficacy and safety of eteplirsen for the treatment of DMD, an NDA should be fileable," and outlined examples of additional data and analysis that, if positive, would be vital to enhance the acceptability of an NDA filing by addressing areas of ongoing concern in the existing dataset.

Based on the Agency's guidance, Sarepta intends to begin several additional trials with eteplirsen later this year in exon-51 amenable genotypes. These studies would include a trial with predefined efficacy endpoints for ambulatory patients between the ages of 7 to 16 years who can walk a minimum distance, and two additional trials that would assess safety and biomarkers in DMD patients younger than 7 years and DMD patients who have advanced in their disease progression to a point they cannot walk a minimum distance or have become non-ambulant.

Furthermore, Sarepta plans to begin a placebo-controlled study with one or more of its follow-on DMD exon-skipping drug candidates by the end of the year.

Sarepta also intends to immediately take actions to initiate the additional eteplirsen studies with the aim of beginning dosing in the confirmatory study in the third quarter, with dosing in the additional trials to begin later this year.