CureDuchenne’s Funding Bears Fruit with Drisapersen: First-Ever New Drug Application (NDA) Accepted by Food and Drug Administration (FDA) For Duchenne Muscular Dystrophy

Sarepta Therapeutics Separately Completes New Drug Application for Eteplirsen

NEWPORT BEACH, Calif., June 29, 2015 – CureDuchenne today applauded the U.S. Food and Drug Administration (FDA), which took the critical step of accepting the first-ever New Drug Application (NDA) for a possible treatment for those who suffer with Duchenne muscular dystrophy. The FDA said it will accept BioMarin Pharmaceutical’s NDA for drisapersen, the first Duchenne drug ever to reach this stage.

Separately, Sarepta Therapeutics has announced the completion of an NDA for its drug eteplirsen, also intended to help Duchenne patients.

CureDuchenne, the national nonprofit dedicated to finding a cure for Duchenne muscular dystrophy, was an early funder and supporter of both these promising treatments.

“The Duchenne community is grateful to BioMarin for their dedication in developing this drug, and CureDuchenne is proud to support the first-ever treatment to reach this milestone in the U.S. It could dramatically improve the quality of life for those with Duchenne,” said Debra Miller, co-founder and CEO of CureDuchenne. “We applaud the FDA on this step and are hopeful for an approval later this year.”

In response to the news from Sarepta, Miller said: “We are thrilled by the progress of eteplirsen. Reaching the NDA stage of the drug approval process is a significant step toward our ultimate goal of curing this disease. The Duchenne community can draw hope from this latest news, and Sarepta and CureDuchenne will continue to build on this success.”

Duchenne muscular dystrophy, an inherited genetic disease with no cure and no treatment up to now, causes progressive muscle degeneration, leaving patients wheelchair-bound by their mid-teens. Almost all of them are boys and young men. Most do not survive their mid-20s.

Drisapersen was developed by Prosensa, which was recently acquired by BioMarin. Both it and Sarepta’s drug are amenable to exon 51 skipping – essentially, ignoring mutations in a patient’s genome that prevent the body from producing a vital protein called dystrophin. Lack of dystrophin is what causes muscle deterioration. Exon skipping alters the splicing pattern of the Duchenne mutation, which encourages the cells to produce new dystrophin, thereby maintaining muscle strength. Exon skipping is a mutation-specific treatment, meaning that it is a form of personalized medicine. This drug, therefore, would positively affect only about 13 percent of those with Duchenne muscular dystrophy.

”We believe strongly that the science behind this research will one day be able to help a larger percentage of those with Duchenne. We will continue to support their efforts in the hopes that promising treatments can become a promise for a cure,” said Miller.

CureDuchenne was founded in 2003 with a focus on saving the lives of those with Duchenne muscular dystrophy, a disease that affects more than 300,000 boys worldwide. With support from CureDuchenne, seven research projects have advanced to human clinical trials, and three of these pharmaceutical treatments could be approved by the FDA within the next year. These treatments may lessen the effects of the disease for those with certain mutations of Duchenne, but there is still much work to be done to find a cure. For more information, please visit and follow us on Facebook, Twitter and YouTube.