CureDuchenne Responds to FDA Advisory Committee’s Recommendation Against Approval of Eteplirsen for Duchenne Muscular Dystrophy


CureDuchenne, the California-based nonprofit organization dedicated to finding a cure for every person with Duchenne muscular dystrophy, has released the following statement from its co-founder and CEO, Debra Miller, following the recommendation against approval by the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) for Sarepta’s exon-skipping drug eteplirsen:

“I’m naturally disappointed by the vote, but I’m grateful that members of the committee were clearly moved by patients’ testimony, and I hope the FDA will note that several appeared to have more problems with the limitations of the studies than with the effectiveness of eteplirsen itself. We are as determined as ever to find treatments for Duchenne muscular dystrophy, and we believe that exon-skipping drugs, perhaps including eteplirsen, will be part of the answer.

“Duchenne manifests throughout the body, affecting the brain, heart, diaphragm, muscles and bones, and is unlikely to be cured by any single drug. This is why we have invested in different types of therapies to address all aspects of the disease.

“Because of the complexity of genetic code, eteplirsen is applicable to about only 13 percent of Duchenne patients; but there are other drugs in the development pipeline that target different genetic mutations that can cause Duchenne.

“Panel members asked some tough questions today, and we appreciate the need for that. But we also believe Duchenne patients need options. We need a variety of possible therapies so that doctors and researchers can determine the best possible combination to fight Duchenne. We worry about the consequences of inaction for those with this disease.

“The courage of the Duchenne community inspires us to continue to work to find new, innovative therapies to help every Duchenne patient. We will not rest until all patients with Duchenne muscular dystrophy have access to treatment and, ultimately, a cure.”

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