CureDuchenne Media Statement Regarding Prosensa Announcement of Regulatory Path Forward for Drisapersen as a Potential Treatment for DMD

Newport Beach, Calif. , June 3, 2014: CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, issued the following statement today regarding the announcement by Prosensa Holding N.V. (NASDAQ: RNA) that the United States Food and Drug Administration (FDA) has outlined a regulatory path forward, under an accelerated approval pathway, for drisapersen, a potential treatment for Duchenne Muscular Dystrophy (DMD).

“This marks a significant milestone and gives hope to the boys and their families living with this devastating disease that a treatment is on the near-term horizon.  It’s been over 10 years since we began our partnership with Prosensa, and although the drug is not yet approved, these results validate our early efforts to fund exon skipping research,” said Debra Miller, CEO and Founder, CureDuchenne. “We applaud Prosensa for their tireless work in finding a treatment for the disease.”

“CureDuchenne has been a loyal and excellent supporter since the very early days of Prosensa.  It is a great example of a collaborative partnership between a patient advocacy group and a biopharmaceutical company. We are very pleased with CureDuchenne’s support over many years,” said Hans Schikan, Chief Executive Officer of Prosensa.

CureDuchenne, a leader in raising awareness and funding research to find a cure for Duchenne, was the first advocacy organization to provide funding to Prosensa in 2004 to help develop exon skipping to treat Duchenne. Since then, CureDuchenne has continued to work closely with Prosensa.

According to Prosensa, following the positive feedback from the FDA, Prosensa has confirmed that it will pursue a New Drug Application (NDA) filing for drisapersen with the FDA, under an accelerated approval pathway based on existing data. Prosensa plans to submit a file later this year and will commit to the initiation of two confirmatory post-approval studies. In conjunction with this, Prosensa said it will continue with plans to re-dose an initial cohort of boys in the third quarter of 2014 who have previously participated in clinical studies with drisapersen.

Currently, there is no treatment for Duchenne, a progressive muscle-wasting disease and the most common and lethal form of muscular dystrophy. Duchenne impacts one in every 3,500 boys; nearly 20,000 boys are living with the disease in the United States. Boys with Duchenne are usually diagnosed before the age of 5 and are in a wheelchair by age 12; most don’t survive their mid-20s.

Click here to see Prosensa’s press release.

About CureDuchenne
CureDuchenne is a national nonprofit organization located in Newport Beach, Calif., dedicated to finding a cure for Duchenne, the most common and most lethal form of muscular dystrophy. As the leading genetic killer of young boys, Duchenne affects more than 300,000 boys worldwide. CureDuchenne has garnered international attention for its efforts to raise funds and awareness for Duchenne through venture philanthropy. With the help of CureDuchenne’s distinguished international panel of Scientific Advisors, funds raised by CureDuchenne support the most promising research aimed at treating and curing Duchenne. To date, seven CureDuchenne research projects have made their way into human clinical trials – a unique accomplishment as few health-related nonprofits have been as successful in being a catalyst for human clinical trials. For more information, visit www.cureduchenne.org.