Whitehouse petition: Urge the FDA to Say YES to Accelerated Approval for children with Duchenne.


Urge the FDA to Say YES to Accelerated Approval for safe, effective therapies for children with Duchenne.

We urge the Food and Drug Administration (FDA) to use the Accelerated Approval pathway for approval and access to safe, effective therapies for Duchenne Muscular Dystrophy - the leading genetic killer of children that impacts 1 out of every 3,500 boys born in the United States. It's time for the FDA to Say Yes and make this the first generation of Duchenne survivors.



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 Eteplirsen was the catalyst for the petition.  The petition is for Accelerated Approval for safe, effective therapies for children with Duchenne.  It does NOT favor any drug over another   

Thank you Jason. I probably read a message that was posted immediately below the announcement for the petition and thought that the message was part of the petition.

Keith, I hope that today's news augurs speedier access to treatments for boys with other compromised exons.

Keith Van Houten said:

Drugs are approved by the FDA on the basis of clinical benefit.  If the biomarker dystrophin is approved as a surrogate endpoint, it will allow faster studies, under the accelerated approval pathway.  Because, you'd be able to get an interim approval based on the surrogate, without waiting to see clinical benefit.  You still have to show clinical benefit in a confirmatory trial to get full approval.  I can't see how surrogate endpoints have anything to do with the size of trials.  Trial size is related to what's required to show statistical significance of the results.

The FDA has watered down the 6MWT, the gold standard to suggest it is only a surrogate!   They should recognize 6 MWT as clinical demonstration and dystrophin as a surrogate!

This should answer your questions on where the FDA standards today.

Sarepta April 21 webcast



The FDA Returns to Its Dark Ages

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