Summary from Seeking Alpha
- Based on updated guidance from the FDA regarding an early approval pathway for eteplirsen, Sarepta Therapeutics (SRPT) plans to file an NDA by the end of 2014.
- The agency provided specific examples of additional safety and efficacy data for Duchenne muscular dystrophy that would enhance the acceptability of the NDA. The company will conduct several open label confirmatory studies later this year on patients with exon-51 amenable genotypes.
- The company plans to conduct three studies: 1) ambulatory patients between the ages 7 and 16 years who can walk a minimum distance, 2) patients younger than 7 years, 3) DMD patients who cannot walk a minimum distance or who are non-ambulant.
- The firm also plans to start a placebo-controlled study with one or more if its follow-on DMD exon-skipping drug candidates by year end.
Please read here for the Sarepta press release: https://investorrelations.sarepta.com/phoenix.zhtml?c=64231&p=irol-newsArticle&ID=1920025&highlight=
CureDuchenne is proud to be an early supporter of Sarepta by providing funding that enabled the company to move forward with their clinical trial of eteplirsen. We look forward to continued success for eteplirsen and all of their additional exon programs.