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Will Sarepta Therapeutics’ Orphan Drug Eteplirsen For Duchenne Muscular Dystrophy Receive Accelerated Approval ? – Perspective

October 24, 2012

       A fascinating October 23, 2012, article titled,”Sarepta Part I: The Possibility Of An Accelerated Approval Of Eteplirsen” discusses whether or not Sarepta Therapeutics’ orphan drug Eteplirsen for Duchenne Muscular Dystrophy (DMD) will receive accelerated approval from the FDA.’s article key points : 

1)  If Eteplirsen receives an accelerated FDA approval, this “would move the drug’s launch timeline 2 years ahead“ & would also “help the company to gain a significant edge over its close competitor, Prosensa/GSK’s Phase III candidate PRO051, in this billion-dollar global market” 

2)  Key catalyst for Sarepta Therapeutics will be the end-of-phase II FDA meeting regarding Eteplirsen’s regulatory pathway in early 2013 

3)  FDA has a history of “approved orphan drugs based on very small trials”. 

References October 15, 2012 article titled,”Duchenne Muscular Dystrophy: Pipeline & Partnering Snapshot”   

Parent Project Muscular Dystrophy October 23, 2012 Article titled,”Prosensa Announces Progress on Exon Skipping Compounds for the Treatment of Duchenne Muscular Dystrophy”  

Prosensa October 23, 2012 Press Release titled,”Prosensa announces progress on Exon Skipping Compounds for the treatment of DMD – milestone payments received from GSK” October 23, 2012 Article titled,”Frustrating Trading Session For Sarepta”.  

Copyright  © 2012-2013, Ann-Teresa Cusenza and Orphan Druganaut Blog.  All rights reserved.

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