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 (GSK) and licensed from Prosensa Therapeutics.
CureDuchenne has worked closely with the FDA to expedite Duchenne drugs through the regulatory process. CureDuchenne provided significant early funding for the development of drisapersen and Sarepta Therapeutics’ eteplirsen, both drugs have shown to replace the missing dystrophin protein and improve the outcomes in clinical trials.
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). The breakthrough therapy designation was based on results from the Phase II study.
The Breakthrough Therapy designation means that the FDA has reviewed the data for drisapersen and will provide additional resources. This classification generally is for clinical programs that demonstrate medical significance.
CureDuchenne has been a longtime supporter of Prosensa. CureDuchenne provided early-stage funding 10 years ago for Prosensa’s antisense (exon skipping) research that led to the development of drisapersen, a promising novel drug for Duchenne. Drisapersen has shown promising results in Phase II clinical trials released in early 2013 and CureDuchenne looks forward to seeing Phase III data later this year.
CureDuchenne was the first muscular dystrophy organization to recognize the potential of Prosensa’s exon skipping research and in 2004 committed significant funds to further that research. A few years later, venture capital firms validated CureDuchenne’s investment with several million dollars to continue their research. In 2009, GlaxoSmithKline committed up to $650 million to put four of Prosensa’s exon skipping compounds on the research and development path.
We applaud the effort of all those who are working on a treatment for Duchenne. We look forward to the day that there is a cure for Duchenne.