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Sarepta Therapeutics’ Orphan Drug Eteplirsen And FDASIA

October 19, 2012

       Fascinating October 17, 2012 article from Elsevier Business Intelligence on Sarepta Therapeutics’ orphan drug Eteplirsen for the treatment of Duchenne Muscular Dystrophy (DMD) and FDA’s new “Breakthrough Therapy” Designation as defined in the new FDA Safety & Innovation Act (FDASIA). 

        Following are a few points made by the article : 

1)  Sarepta Therapeutics is valued at “almost $650 million, about 10 times higher than it was when the year began” 

2)  Is the most recent data from the 48-week Eteplirsen study enough to allow for FDA “accelerated approval” right away ? 

3)  Under the new “accelerated approval” provisions from FDASIA signed by President Obama in July 2012, the FDA is encouraged to consider the pathway for drugs to treat rare diseases. Can this new definition of “accelerated approval” be applied for Eteplirsen for DMD ? 

4)  But there is another new way under FDASIA, called the “Breakthrough Therapy” Designation. Will or has Sarepta Therapeutics already filed using the “Breakthrough Therapy” Designation for Eteplirsen for DMD ?  

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

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