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Hopes high for muscular dystrophy drug

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By The Washington Post
Monday, April 21, 2014, 10:03 p.m.

A new drug designed to slow the progression of Duchenne muscular dystrophy, a devastating disease that overwhelmingly affects boys and typically leaves them needing wheelchairs by their teens and dead in their 20s, has received a long-awaited boost from the Food and Drug Administration.

Massachusetts-based Sarepta Therapeutics said Monday that FDA officials detailed a potential path forward for the drug, eteplirsen, and indicated a willingness to consider it for accelerated approval. The news puts eteplirsen one step closer to becoming the first approved treatment for Duchenne, a disease that affects about one in 3,500 boys globally.

“This provides the opportunity to get the drug approved and in the hands of all the boys who can benefit from it sometime in 2015,” Sarepta chief executive Chris Garabedian said. “I think that's a huge win for the [Duchenne] community and for Sarepta.”

The move follows a tumultuous year in which the FDA initially appeared headed toward swift approval of eteplirsen based on promising results from a trial involving 12 young boys, whose muscular deterioration seemed virtually halted by the drug. But the agency hit the pause button in November, saying it would be “premature” for Sarepta to seek approval of its drug with the failure of a separate trial involving a different Duchenne treatment that used similar technology.

Months of behind-the-scenes deliberations followed over the effectiveness of eteplirsen, as well as an aggressive public campaign waged by patients' families determined to keep pressure on the agency.

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