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Sarepta Therapeutics And The FDA: Putting The Pieces Together

April 16, 2013

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 orphan drug Eteplirsen for Duchenne Muscular Dystrophy (DMD). Will the company file for Accelerated Approval or Breakthrough Therapy Designation ? What were the results of the recent End-of-Phase II meeting between Sarepta Therapeutics and the FDA’s Division of Neurology Products in March ?

Also, Sarepta Therapeutics has recently attracted attention and interest due to several April events :

•  Sarepta Therapeutics’ update on April 5 on the Eteplirsen data results from Study 202 (Phase IIb open-label extension study) for the Walking Test through 74 Weeks

•  Strong presentation given by Chris Garabedian, President and CEO of Sarepta Therapeutics, at last week’s World Orphan Drug Congress USA in Washington D.C.

•  Sarepta Therapeutics’ announcement on April 11 that the company has entered an exclusive, worldwide licensing agreement with the University of Western Australia (UWA), for intellectual property rights to support development of exon-skipping drug candidates for the treatment of DMD.

Finally, some of the pieces of the puzzle are coming together. 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 later this quarter. The End-of-Phase II CMC (Chemistry, Manufacturing, and Controls – part of the pharmaceutical product application to the FDA) meeting with the FDA is now expected for the 3rd quarter 2013.

According to’s Adam Feuerstein, the FDA is “actively reviewing the efficacy and safety of Eteplirsen even though the drug has not been formally filed for approval yet.”


FDA Definition of Accelerated Approval

Sarepta Therapeutics April 15, 2013 Conference Call

Please Note: “Jigsaw” by User: Amada44 (Own work) [Public domain] | Wikimedia Commons.

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

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