Sarepta to file eteplirsen NDA by year end

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.

Shares are up 78% premarket on heavy volume.

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Comments (5)
  • Hoang6
    , contributor
    Comments (358) | Send Message
    Just a plan to file an NDA (by the end of the year) makes the stock soar 78% is laughable. Nowadays, things are getting ridiculous!
    21 Apr 2014, 12:07 PM Reply Like
  • rob1159
    , contributor
    Comments (9) | Send Message
    What's ridiculous, bordering on obscene, is the FDA's bureaucratic nonsense in not giving these kids a chance on a medication that if nothing else has a very high safety profile. I don't know how they sleep at night.
    22 Apr 2014, 08:53 PM Reply Like
  • SharkDude
    , contributor
    Comments (785) | Send Message
    Exactly. The market should kill companies who have inferior product. But the FDA kills small biotech before they get a chance to prove themselves. That I snot their job.
    23 Apr 2014, 12:52 AM Reply Like
  • kingry
    , contributor
    Comments (33) | Send Message
    Does the NDA come after phases 1 2 and 3 of clinical trials or before?
    21 Apr 2014, 12:50 PM Reply Like
  • rcassidyusa
    , contributor
    Comment (1) | Send Message
    The stock I bought for 28+ closed for 24.40 Friday, and the 30 APR calls that I sold for more than 280, expired: today I sold for 35+, 40% 1.5 mo later.
    Yes Hoang6, it IS laughable, and I AM laughing!
    22 Apr 2014, 02:21 AM Reply Like
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