CureDuchenne’s Early Funding Projects Take a Big Step Forward Today

FDA Accepts NDA for Drisapersen, the First-Ever Treatment for Duchenne Muscular Dystrophy to Reach this Milestone in the U.S., and Sarepta Therapeutics Completes NDA Submission to FDA for Eteplirsen 

It is a big day for the Duchenne community.  It is also a proud moment for CureDuchenne. 

Today, BioMarin announced that the U.S. Food and Drug Administration (FDA) has accepted for review its submission of a New Drug Application (NDA) for drisapersen for the treatment of Duchenne muscular dystrophy.  Separately, Sarepta Therapeutics has also announced the completion of an NDA for its drug eteplirsen.  CureDuchenne was an early funder and supporter of both these promising treatments.

In 2004, when a tiny Dutch biotech company, Prosensa, needed funding to develop a drug, now called drisapersen, the only organization to step up and provide support was CureDuchenne.  Again, 10 years later when Prosensa needed funding to provide this drug to the many boys who had been in the drisapersen trials and to continue to work on additional exon skipping programs, CureDuchenne was the only organization to commit significant funding.

In 2010, CureDuchenne collaborated with Children’s National Medical Center and the Foundation to Eradicate Duchenne to provide early funding and expertise to enable Sarepta (then AVI Biopharma) to move forward with the development of eteplirsen.

We are delighted at word of today’s milestones.  And we are pleased to see other organizations take notice of drisapersen since BioMarin acquired Prosensa earlier this year and, we are thrilled that BioMarin has committed such significant resources to develop drisapersen.  We have had the pleasure of getting to know their team and we are so impressed with their dedication to Duchenne patients. At CureDuchenne we will continue our work to get these drugs approved, as well as all drugs that may improve the lives of those with Duchenne.

For years, hundreds of thousands of children around the world with this genetic and deadly disease have had nothing to look forward to in terms of a cure or treatment.  Patients with Duchenne are diagnosed in early childhood, use a wheelchair by their mid-teens and often have a life expectancy of their mid-20s.

Duchenne causes muscular deterioration through the lack of dystrophin that inevitably makes muscle regrowth impossible.  Both drisapersen and eteplirsen are amenable to exon 51 skipping and alter the splicing pattern of the Duchenne mutation, encouraging new cellular dystrophin production to maintain muscular strength.

The Duchenne community is grateful to BioMarin and Sarepta for their dedication in developing these drugs, and to the hundreds of boys who made significant sacrifices to participate in the trials. CureDuchenne is proud to support all treatments that could dramatically improve the quality of life for those with Duchenne.  We applaud the FDA and the pharmaceutical companies.

CureDuchenne will continue supporting Duchenne research, hopeful that it will one day help the more that 300,000 people worldwide beat Duchenne and give them hope in living long and productive lives.