Sarepta Therapeutics To File NDA For Muscular Dystrophy Drug
Sarepta Therapeutics, a company focused on developing RNA-based treatments, announced its intent to file a New Drug Application for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) by the end of 2014.
Eteplisen is the company’s lead exon-skipping drug candidate under development for the treatment of patients with DMD who have a genotype disposed to skipping of exon 51.
The company said it reached the decision to file an NDA by the end of the year based on a guidance letter from the FDA. According to the agency, an NDA for eteplirsen could be filed under a potential Accelerated Approval pathway. The FDA also provided guidance on an open-label, historically controlled confirmatory study of the drug as well as initial guidance on a placebo-controlled study on one or more of the company’s follow-on DMD drug candidates.
Chris Garabedian, president and CEO of Sarepta Therapeutics, said, “As we announce our plan to submit an eteplirsen NDA by the end of 2014, we are very pleased with the detailed guidance that the FDA has provided us on a potential eteplirsen approval pathway and their support of a historically controlled eteplirsen confirmatory study. We also appreciate that the FDA shares our urgency in dosing a broader base of eteplirsen patients and has encouraged us to begin the clinical program with our follow-on exon-skipping drugs as soon as possible.”
Based on the FDA’s guidance, Sarepta announced that it plans to launch several additional clinical studies with eteplirsen later this year in exon-51 amenable genotypes.
Edward Kaye, SVP and CMO of Sarepta Therapeutics, said, “We are excited to have guidance from the FDA that allows us to move quickly into additional clinical trials with eteplirsen to confirm our current understanding of eteplirsen’s safety profile, its effect on dystrophin production, and its impact on clinical outcomes in DMD patients. We are particularly pleased that the FDA shares our interest in accelerating the clinical development of our follow-on exon-skipping drugs and we expect to initiate enrollment in this trial later this year.”