CureDuchenne Comments on U.S. Food and Drug Administration Breakthrough Therapy designation for GlaxoSmithKlineÌ¢‰â‰ã¢s/Prosensa Therapeutics’ drisapersen for Treatment of Patients with Duchenne Muscular Dystrophy

Newport Beach, Calif., June 27, 2013 – CureDuchenne is pleased that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to drisapersen, an exon-51 skipping compound for the potential treatment of patients with Duchenne Muscular Dystrophy. Drisapersen is being developed by GlaxoSmithKline plc (GSK) and licensed from Prosensa Therapeutics.

The Breakthrough Therapy designation is one of several programs created by the FDA to expedite the development and review of drugs for serious or life-threatening conditions. It was enacted in 2012 as part of the Food and Drug Administration Safety and Innovation Act (FDASIA). For more information on the Breakthrough Therapy designation, click here.

“The Breakthrough Therapy designation means the FDA has reviewed the data for drisapersen and will provide additional resources,” said Debra Miller, CEO and Founder, CureDuchenne. “This classification generally is for clinical programs that demonstrate medical significance. Duchenne is a disease that progresses very quickly and for which there’s currently no cure, so on behalf of the Duchenne community, we are grateful to the FDA for recognizing the need and potential for this drug. We hope to see the drug approved soon.”

CureDuchenne, a leader in raising awareness and funding research to find a cure for Duchenne, committed significant funds at an early stage to Prosensa’s exon skipping research that led to the development of drisapersen. CureDuchenne has worked with the FDA to help expedite approval of Duchenne drugs based upon the severity of the disease and the risk tolerance of the patient community. 

“These results validate our early efforts to fund novel research and offer hope for finding an effective and safe treatment for all those living with Duchenne,” Ms. Miller continued. “We applaud GlaxoSmithKline and Prosensa for their tireless work in developing drisapersen as a treatment for the disease.”

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.

About CureDuchenne

CureDuchenne is a national nonprofit organization 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, the organization has raised more than $11 million towards promising research aimed at treating and curing Duchenne. In fact, seven CureDuchenne research projects have made their way into human clinical trials – a unique accomplishment as few health-related nonprofits have been successful in being a catalyst for human clinical trials. For more information on how to help raise awareness and funds needed for research, visit www.cureduchenne.org.