US FDA clears pathway for Sarepta’s eteplirsen filing by year end

US RNA-based drug developer Sarepta Therapeutics (Nasdaq: SRPT) says it plans to submit a New Drug Application to the US Food and Drug Administration by the end of 2014 for the approval of eteplirsen for the treatment of Duchenne muscular dystrophy (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. However, last fall, the FDA said it considered the company’s filing for the drug to be premature, and leading Sarepta’s share to tank (The Pharma Letter November 12, 2013). However, in early trading this morning, Sarepta’s shares rocketed 60% to $39.00.

The new plan to submit an NDA for eteplirsen by the end of 2014 is based on a guidance letter from the agency that proposed a strategy regarding the submission 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 FDA stated 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, will be important to enhance the acceptability of an NDA filing by addressing areas of ongoing concern in the existing dataset.