CureDuchenne Celebrates Submission of First-Ever New Drug Application for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping; CureDuchenne Was Early Funder of Drisapersen’s Development
BioMarin Pharmaceutical has submitted a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) – the first ever for a possible therapy for Duchenne muscular dystrophy amenable to exon 51 skipping– for its drug drisapersen. No other Duchenne therapy has reached this stage before in the United States.
Duchenne is a muscle-wasting genetic disorder that affects more than 300,000 boys and young men worldwide, and drisapersen could become the first Duchenne therapy approved by the FDA.
Development of drisapersen was made possible, in part, by investments of $2 million by CureDuchenne and CureDuchenne Ventures. CureDuchenne has supported Prosensa, the original developer of drisapersen, since the company’s inception, and has helped it at critical times in the development of new drugs. BioMarin acquired Prosensa in late 2015.
Duchenne affects 1 in 3,500 boys. They are usually diagnosed by the age of 5 and in a wheelchair by age 12. Most don’t survive their mid-20s. Because of a genetic variant, Duchenne patients are missing a key muscle protein called dystrophin. Without it, muscle cells become damaged and die. The simplest of tasks become difficult for those with Duchenne, and in the later stages, heart and breathing muscles begin to fail.
“We are filled with hope as the first potential therapy for Duchenne muscular dystrophy reaches this important juncture,” said Debra Miller, co-founder and CEO of CureDuchenne. “We have been working toward this goal for more than a decade. We are grateful to BioMarin for the commitment it has made to help the boys and young men with this disabling condition. And we all agree that we cannot rest until there is a cure.”
The exon-skipping method used in drisapersen causes cells to “skip” over faulty sections of genetic code that prevent the formation of dystrophin. Researchers say the result should be shorter but functional proteins, transforming Duchenne muscular dystrophy into a much milder disease.
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. 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 advanced into human clinical trials – a unique accomplishment as few health-related nonprofits have been successful in being a catalyst for human clinical trials.