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The Power Of Patient Advocacy Groups On Orphan Drug Development: Sarepta Therapeutics’ Orphan Drug Eteplirsen For Duchenne Muscular Dystrophy

November 6, 2012

       Several patient advocacy groups have met with the FDA to help Sarepta Therapeutics obtain Accelerated Approval for orphan drug Eteplirsen, for the treatment of Duchenne Muscular Dystrophy (DMD) : 

1)  Parent Project Muscular Dystrophy (PPMD) has had 2 meetings with FDA’s Neurology Division 

2)  The Duchenne Alliance submitted a “dossier to FDA last week to get DMD included on a list of rare diseases eligible for special considerations, including accelerated access and approval, under the recently approved “PDUFA V” legislation …” 

3)  Cure Duchenne has already met with the FDA & is organizing a webinar later in November 2012 where DMD families can ask questions of the FDA. 

Key Upcoming Catalyst Events 

1)  Sarepta Therapeutics October 30, 2012 Press Release Announcing 3rd Quarter 2012 Financial Results & Corporate Update for Wednesday, November 7, 2012 

2)  Action Duchenne 10th International Conference in London, November 9 – 10, 2012 with Dr. Ed Kaye (AVI BioPharma US/Sarepta Therapeutics) presenting results of the Phase 2B Eteplirsen 48-Week Extension Trial on Saturday, November 10, 2012    

3)  Sarepta Therapeutics November 6, 2012 Press Release announcing Company Overview Presentation at the 2012 Lazard Capital Markets 9th Annual Healthcare Conference for Tuesday, November 13, 2012 

4)  Eteplirsen Phase IIb study results to be presented by Sarepta Therapeutics to the FDA in early 2013; Also, Sarepta Therapeutics is to ask for permission to file for Accelerated Approval. 

References 

**    The Duchenne Alliance November 4, 2012 Document titled, ”Duchenne Muscular Dystrophy as a Model Disease for PDUFA-V”  

TheStreet.com November 6, 2012 article titled, ”Advocates, Patients Rally to Support Sarepta Early Drug Approval Push”  

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

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