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Sarepta Therapeutics Announces Recent Corporate Developments For Orphan Drug Eteplirsen

November 9, 2012

       Sarepta Therapeutics announces on November 7, 2012, recent corporate developments for orphan drug Eteplirsen for the treatment of Duchenne Muscular Dystrophy (DMD) : 

–  Presents preclinical safety data showing Eteplirsen is well tolerated in 9-month repeat dose toxicity evaluation in cynomolgus monkeys at the 8th Annual Oligonucleotide Therapeutic Society (OTS) October 2012 meeting. Reference Page 78 of the Abstract Book  

– Presents 48-week data from Phase IIb study evaluating Eteplirsen for treatment for DMD boys at the 17th Annual International World Muscle Society October 2012 Meeting in Perth, Australia 

– Announces treatment of Eteplirsen met primary efficacy endpoint at 48-weeks & achieves significant benefit on the 6-minute walk test (6MWT) 

–  In process of putting together a briefing document for the FDA for the Eteplirsen study results. Will request a meeting by the end of 2012 to potentially setup a meeting with the FDA in the 1stQ 2013 (it is not scheduled yet). One goal of the FDA meeting is to review the “design of a confirmatory pivotal study & acceptable endpoints for full approval of Eteplirsen …. also hope to better understand the feasibility of an accelerated approval filing on the studies we’ve conducted to date”.  

–  Sarepta Therapeutics is not “ … seeking a partner to help … with Eteplirsen in the US. We have stated our willingness to consider a partner for ex-US or ex-North American rights … “.   


Orphan Druganaut November 8, 2012 Blog Post titled, ”The power of patient advocacy groups on orphan drug development: Sarepta Therapeutics’ orphan drug Eteplirsen for Duchenne Muscular Dystrophy” November 7, 2012 Transcript of Sarepta Therapeutics’ CEO discussing Q3 2012 Results & DMD Program  

Copyright © 2012-2013, Orphan Druganaut Blog.  All rights reserved.

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