CureDuchenne, the leading nonprofit organization that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, is pleased by today’s announcement by Sarepta Therapeutics (formerly AVI BioPharma) that its exon-skipping compound, eteplirsen, achieved significant clinical benefit after 48 weeks of treatment in a Phase IIb study in Duchenne muscular dystrophy. CureDuchenne, along with Children’s National Medical Center in Washington, DC and the Foundation to Eradicate Duchenne, provided funding in 2010 for this research to progress into human clinical trials.
Eteplirsen helped increase the production of dystrophin, which is lacking due to a defect on the dystrophin gene that causes Duchenne, and slowed the progression of the disease, as measured by the 6-minute walk test. This is a milestone for the company and patients alike. The result will pave the way for an end of phase II meeting between the company and the FDA to discuss and agree to the plan for a phase III registration trial. Sarepta will scale up the manufacturing to accommodate a larger trial.
“This is an important day for Duchenne,” said Chris Garabedian, CEO of Sarepta Therapeutics. “We are grateful to CureDuchenne, and other organizations, for their support and funding that allowed us to proceed this far in the clinical trial process and move a step closer to finding a treatment for Duchenne.”
“We have personally seen the positive impact this drug has had on our son,” said Jenn McNary who has two sons with Duchenne. Ten-year-old Max is one of the boys enrolled in the Sarepta trial, but his 13-year-old brother Austin was not eligible for the trial. “Max has shown significant improvement and is stronger, while Austin is getting weaker. We’re hoping these results will allow Sarepta to get approval quicker so other Duchenne boys can benefit from this treatment that helps slow the progression of the disease.”
As the most common and lethal form of muscular dystrophy, Duchenne impacts 1 in 3,500 boys. Boys with Duchenne are usually diagnosed before the age of 5, in a wheelchair by age 12 and most don’t survive their mid-20s.
“This is very encouraging news for the Duchenne community that a treatment is within our reach,” said Debra Miller, founder and CEO of CureDuchenne. “CureDuchenne is committed to continuing to fund research, like we did for the Sarepta trial, until a cure is found that helps all Duchenne boys. We are hopeful, but also must remain cautious until treatments are FDA-approved.”