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What Additional Data Did Sarepta Therapeutics Present For Orphan Drug Eteplirsen (December 7, 2012) ?

December 7, 2012

       On December 7, 2012, Jerry R. Mendell, MD, from Nationwide Children’s Hospital in Columbus, Ohio, gives a presentation titled, “Results at 62 weeks of a Phase IIB Extension Study of the Exon-Skipping Drug Eteplirsen in Patients with Duchenne Muscular Dystrophy (DMD)”, at the European Neuromuscular Centre (ENMC) Workshop in Naarden, Netherlands. 

                  The Orphan Druganaut Blog leaves it to the reader to view the presentation for the science behind orphan drug Eteplirsen, the Eteplirsen Phase IIB 62-week 6MWT results, and the Phase IIB Extension Study safety data results.

                 To briefly summarize : 

–  Eteplirsen maintains a “statistically significant clinical benefit on the primary clinical outcomes, the 6-minute walk test (6MWT), over a placebo/delayed treatment cohort through week 62 ….” 

–  Eteplirsen is well tolerated with no clinical treatment-related adverse events”. 

       Upcoming Sarepta Therapeutics catalyst events

–  1st Quarter 2013 => Submission of briefing documents to FDA for an end-of-Phase II Meeting, including querying whether or not Eteplirsen is appropriate for Accelerated Approval 

–  2014 => A confirmatory study for Eteplirsen to start. 

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

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