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Refining A Controversial Endpoint: Sarepta Banking On Dystrophin Levels For Golodirsen Approval In DMD

 
• By Michael Cipriano

While glodirsen did show higher levels of dystrophin production than eteplirsen did, increase is still unlikely to satisfy US FDA reviewers who disagreed with Janet Woodcock's decision to grant accelerated approval to eteplirsen.

Sarepta Therapeutics Inc. is hoping to bring another Duchenne muscular dystrophy (DMD) product through the US FDA by relying on a controversial surrogate endpoint, and question now is what kind of precedent will the company's golodirsen candidate set, as the company announced plans to complete a rolling submission of a new drug application (NDA) by the end of 2018 based on increase in mean dystrophin protein levels.

The March 12 announcement comes as Sarepta said it received the minutes from Type C meeting with FDA's Division of...

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