Treatments on the Horizon

The Duchenne community is celebrating another promising step toward an approved treatment. On May 19 Sarepta Therapeutics announced plans to submit a rolling NDA for eteplirsen with the final NDA submission landing in the FDA’s hands by mid-year.

CureDuchenne is proud to be part of the support and funding for eteplirsen. We collaborated with Children’s National Medical Center and the Foundation to Eradicate Duchenne in 2010 giving Sarepta Therapeutics (then called AVI Biopharma) the resources needed to move into clinical development of eteplirsen.

This is the second drug submitted to the FDA in the last month. In late April BioMarin (which acquired Prosensa earlier this year) submitted a rolling NDA for drisapersen. CureDuchenne was an early supporter of exon skipping and funded Prosensa, the original developer of drisapersen, from the beginning. We are proud to say we have provided funding for this drug at critical development times.

The fact that there are two Duchenne drugs in the midst of the FDA approval process is quite a blessing. When our son was diagnosed with Duchenne muscular dystrophy 12 years ago, not only were there no treatments, there was no hope that a treatment was coming. That motivated us to fund impactful research. Potential treatments are now on the horizon, and we are hopeful this is only just the beginning.  

To encourage other biotech companies to continue their research we are now working to help them understand the natural history of Duchenne. This is important as they present their data and design the best clinical trials.

One such example is a key project being developed by Dr. Susan J. Ward in partnership with Dr. James Signorovitch of Analysis Group Inc. CureDuchenne provided seed funding, expertise and resources to help launch this project. The collaboration includes a broad consortium of drug companies, academic institutions and other Duchenne organizations. We will keep the community apprised as this moves forward.

We need to keep our kids with Duchenne ambulatory and healthy as long as possible so that they will be ready to take advantage of these medications once they are approved. That’s why CureDuchenne Cares is working with parents and local PTs around the country to educate and train them on best standards of care. This program is made possible through educational grants from our sponsors BioMarin, PTC Therapeutics, Sarepta and Lilly.

CureDuchenne’s goal is to make life-saving treatments possible for Duchenne patients as quickly as possible. With treatments on the horizon, we all share a renewed hope for the future.