Duchenne Community: Let Your Voice Be Heard!


This week the FDA accepted Sarepta Therapeutics’ New Drug Application for eteplirsen with priority review. This follows on the June FDA’s acceptance of BioMarin’s NDA for drisapersen, also with priority review.

The next step for both of these drugs is an FDA Advisory Committee meeting in November where both companies will demonstrate why these drugs should be approved. Duchenne families have the opportunity to participate in this meeting. In fact, it’s vital for us to rally around this pivotal event and make our voices heard.

CureDuchenne was the lead organization to support the development of drisapersen and, CureDuchenne, along with Children’s National Medical Center and Foundation to Eradicate Duchenne, provided crucial funding to Sarepta (then AVI) to enable the development of eteplirsen.  We are proud to be the catalyst for exon skipping and, like you, are hopeful for FDA approval of these drugs soon.

As you know, these drugs will only address 13% of those affected with Duchenne, and we still need drugs to address cardiac and other issues with this disease.  There is more work to be done. But first we need to tell the FDA that the Duchenne community wants drisapersen and eteplirsen.

There are many ways to communicate with the FDA. Please email me at debra@cureduchenne.org if you would like to part of this historic event.

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