Novel Effort Delivers Failed DMD Drug to Former Clinical Trial Participants

When BioMarin failed to win U.S. Food and Drug Administration approval for its experimental Duchenne muscular dystrophy drug drisapersen, it decided to its cease development. Though there were concerns about both the safety and efficacy of the drug, there were patients who participated in the clinical trials who felt that they benefitted from it. CureDuchenne established CD Access, a new nonprofit that negotiated a novel agreement with BioMarin to get control of the existing supply of drisapersen, which the nonprofit is distributing to patients in Canada who participated in the clinical trials. We spoke to Debra Miller, CEO and founder of CureDuchenne and CD Access, about the program, how it works, and whether it serves as a model for getting other similar drugs to patients in the future.