Sarepta Therapeutics announced today plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the first half of 2014 for the approval of eteplirsen, an exon skipping compound under development for the treatment of Duchenne muscular dystrophy. CureDuchenne, a leader in raising awareness and funding research to find a cure for Duchenne, collaborated with Children’s National Medical Center and the Foundation to Eradicate Duchenne in 2010 to provide funding and expertise to enable Sarepta (then AVI Biopharma) to move forward with the development of eteplirsen.
“We continue to be encouraged by the progress of eteplirsen through the clinical trial and FDA approval process,” said Debra Miller, CEO and Founder, CureDuchenne. “Sarepta’s intent to submit an NDA application in the first half of 2014 brings hope to the Duchenne community that we are that much closer to a treatment for a meaningful portion of those suffering with the disease.”
The NDA application is the vehicle through which drug sponsors formally propose that the FDA approve a new pharmaceutical for sale and marketing in the U.S. The data gathered during the animal studies and human clinical trials of an Investigational New Drug (IND) become part of the NDA and typically provide enough information for the FDA reviewers to reach a decision to approve or not approve a drug.
“Given the rapidly progressive nature of the disease, the Duchenne community has always been willing and open to taking risks in order to speed new therapies to those with the disease. The development of eteplirsen has been a great example of international collaboration between scientists, foundations, and Sarepta to share the risk together and make this happen,” said Eric P Hoffman, PhD Director, Research Center for Genetic Medicine, Children’s National Medical Center.
Duchenne is 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.
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.