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MDA Chief Medical and Scientific Officer Testifies at Landmark FDA Hearing for Duchenne Drug

MDA expresses enthusiasm for eteplirsen,
says treatment options need to shift from goal to reality

CHICAGO, April 25, 2016 — Speaking today before an FDA advisory committee, MDA’s chief medical and scientific officer expressed optimism that the drug eteplirsen could change the course of Duchenne muscular dystrophy and be the first of what MDA hopes will be many new treatments for MDA families.

“All of us at MDA, as well as our sister organizations, scientific community, families and supporters, have been working tirelessly to see a time like the present—a time when therapies could be more than just a hope for the future,” MDA Chief Medical and Scientific Officer Valerie Cwik, M.D., told the committee. “We are all here for those living with Duchenne, and the people who love them.”

The FDA convened its advisory committee meeting to review the drug eteplirsen under development by Sarepta Therapeutics.  Eteplirsen is an “exon skipping” drug in late-stage development for the treatment of DMD. Administered by intravenous infusion, it targets a section of genetic code called “exon 51” in the dystrophin gene. If approved by the FDA, it would be the first approved treatment designed to target the underlying cause of the disease.  

MDA has been central to the development of the exon skipping approach from the beginning in the 1990s, having funded foundational work upon which the strategy was built as well as extensive research into the strategy since that time. MDA supported the early development of eteplirsen via funding to Steve Wilton at the University of Western Australia in Perth.

“MDA has led the search for treatments and cures for Duchenne for more than half a century, and will continue to do so until there is a cure,” added Cwik. “Twenty years ago we funded foundational exon skipping research and follow-on studies that led to the development of eteplirsen. And while not a cure, the data indicate that the drug could slow disease progression.” 

Cwik closed her testimony by urging the Advisory Committee to consider all of the tools available to the FDA to allow the widest and earliest access to eteplirsen. While acknowledging the FDA is ultimately responsible for regulatory approval, she referenced overwhelming support from Duchenne scientific and clinical leaders, and DMD families; the urgent and unmet medical need; and the strong safety data.

“It is time that treatment options shift from being a goal to being reality,” concluded Cwik.

Today’s hearing marks one of the final steps in the regulatory review process. The Prescription Drug User Fee Act (PDUFA) action date by which the FDA must make a decision on whether or not to approve eteplirsen is May 26, 2016.

Click here to read the full transcript of Dr. Cwik’s testimony.

About MDA
            MDA is leading the fight to free individuals — and the families who love them — from the harm of muscular dystrophy, ALS and related muscle-debilitating diseases that take away physical strength, independence and life. We use our collective strength to help kids and adults live longer and grow stronger by finding research breakthroughs across diseases; caring for individuals from day one; and empowering families with services and support in hometowns across America. Learn how you can fund cures, find care and champion the cause at