A note from Debra

Sarepta to File Eteplirsen NDA by Year End

Posted on April 21, 2014 12:44 pm

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:  http://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.

Eteplirsen, Drisapersen, the FDA and Dedicated Duchenne Moms

Posted on March 26, 20145:46 pm

Congratulations to the Race to Yes organizers (Tracy Seckler, Jenn McNary, Christine McSherry, Mindy Leffler, and many others) and the Duchenne community for their passionate and successful campaign to petition the White House to urge the Food and Drug Administration (FDA) to use  the Accelerated Approval pathway for approval and access to safe, effective therapies Read more »

Drisapersen Update with Prosensa

Posted on March 19, 20146:13 pm

Prosensa will present a status update on Drisapersen and its follow-on compounds in a patient focused webinar hosted by United Parent Projects Muscular Dystrophy (UPPMD) on Tuesday, March 25 at 1 p.m. ET. If you have any questions regarding recent Prosensa/drisapersen updates or any other topic related to the company’s Duchenne portfolio that you would Read more »