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DMD Race For First FDA Approved Drug: Sarepta Therapeutics Business Developments

June 17, 2013

This is the third in a three part series on recent business developments for the three competitors and their orphan drugs in the Duchenne Muscular Dystrophy (DMD) space :

•   PTC Therapeutics’ Ataluren
•   GlaxoSmithKline (GSK)/Prosensa’s Drisapersen
•   Sarepta Therapeutics’ Eteplirsen.

DMD patients and their families, investors, and the media, have been waiting and debating since the beginning of 2013, as to what Sarepta Therapeutics will announce for their FDA filing plans for Eteplirsen. Will the company file for Accelerated Approval or Breakthrough Therapy Designation ? What are the results of the recent End-of-Phase II meeting between Sarepta Therapeutics and the FDA’s Division of Neurology Products in March 2013 ?

Sarepta Therapeutics provides on April 15, 2013, an update on the company’s discussion with the FDA regarding a potential application for Accelerated Approval of Eteplirsen. The FDA has asked for additional information from the existing Eteplirsen dataset to “inform a decision on the acceptability of this dataset for a New Drug Application (NDA) filing under the Subpart H Accelerated Approval regulatory pathway.” Also, Sarepta Therapeutics receives feedback on :

•  The acceptability of dystrophin as a surrogate endpoint that would reasonably predict clinical benefit in DMD patients

•  The acceptability of the Eteplirsen safety database for a Subpart H Accelerated Approval filing.

As a result of the End-of-Phase II meeting in March, the FDA has asked for two written summaries :

•  A summary to support dystrophin as a surrogate endpoint

•  A detailed discussion of all clinical outcomes in the Eteplirsen study.

Per the March meeting minutes, “The Agency stated that they had not made a final decision regarding acceptability of the proposed Subpart H (Accelerated Approval) NDA filing, and the Agency would consider the additional data submitted by the sponsor before making a final decision.” Sarepta Therapeutics is in the process of submitting both the dystrophin and clinical outcomes summaries to the FDA, and will be asking for a follow-up meeting with the FDA.

Concerns have been expressed about whether Eteplirsen will receive FDA accelerated approval. If Eteplirsen doesn’t receive this, Sarepta Therapeutics may need to move forward with a Phase III trial. In the most recent presentation, CEO Chris Garabedian on May 30, 2013, at the 38th Annual dbAccess Health Care Conference did not provide any definitive positive news as to the fate of the FDA Accelerated Approval designation for Eteplirsen:

… we’ve been focused on preparing these two summary papers that the FDA requested; one on dystrophin, as a surrogate marker in Duchenne Muscular Dystrophy; and the other one on our clinical outcomes from our Phase IIb program … So it looks like that the meeting that we were hoping to have calendered by the end of June, it looks like that meeting would be calendered in the third quarter …..”.

Also, at this presentation, Mr. Garabedian answered a question on whether the company has applied for the FDA Breakthrough Therapy designation for Eteplirsen:

“ … we have not applied for breakthrough status … we are trying to really understand the benefits to us of having a breakthrough designation. We have Fast Track designation which confers many of the same benefits … we want to get further clarity on how they view our dystrophin and our clinical data set because as you know, breakthrough is based on a clinical signal of efficacy.” 

On June 19, 2013, Sarepta Therapeutics is scheduled for a webcast at the Wells Fargo Securities 2013 Healthcare Conference in Boston.


FDA Definition of Accelerated Approval

SeekingAlpha’s Sarepta Therapeutics May 30, 2013 Conference Call Transcript.

Please Note: “View from starting block of a competition swimming pool” by Rufino Uribe [CC-BY-SA-2.0] | Wikimedia Commons.

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

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