MILDRED O COX
2464 Vesta Lane
Bonifay, FL 32425
May 1, 2016
My grandson was diagnosed with Duchenne Muscular Dystrophy at age five. Duchenne is a rare and always fatal disease that impacts one in every 3,500 live male births in the U.S. It is the number one genetic killer of children. It is 100% fatal. There is currently no FDA approved treatment or cure.
Our laws establish an accelerated approval track for promising therapies that meet three main criteria: treats a serious condition, addresses an unmet medical need, and reasonably likely to lead to clinical benefit. Eteplirsen is such a drug being sponsored for fast track approval that has shown to be effective and deserves approval by the FDA.
Thirteen of the world’s leading Duchenne scientists and clinicians participated in the Open Public Hearing at the AdComm meeting of the FDA on April 25
. Each of them stated that based on their extensive experience specifically with Duchenne and in their expert opinion, eteplirsen is effective and deserves approval.
Although this is an emotional topic for parents and families affected by DMD, it is not about emotion. It is about science.
In the 1990’s, the AIDS community demanded faster approval for life-saving medicines. One drug was reviewed and approved in less than two weeks. Thanks to a 2012 law called FDASIA (Food and Drug Safety and Innovation Act) the FDA has even more tools to get drugs to dying patients faster. The FDA just has to use them.
May 26 the FDA will meet to discuss eteplirsen again. Please support the Duchenne community in trying to have eteplirsen made available for our boys. Google “Race to Yes” for more information on how you can help.