Duchenne muscular dystrophy (DMD) advocacy groups are mobilizing to support efforts by Sarepta Therapeutics (SRPT) to seek accelerated approval for its experimental drug eteplirsen. Some of these groups, accompanied by parents of DMD patients, have already met with U.S. Food and Drug Administration officials to make sure regulators understand the urgent need for new therapies. More meetings with U.S. regulators are planned. Sarepta will meet with the FDA, likely early next year, to present results from the eteplirsen phase IIb study and request permission to file for accelerated approval. The company is holding a conference call Wednesday in conjunction with third-quarter financial results. “We are absolutely going to do everything we can to get eteplirsen approved,” said Sharon Hesterlee, senior director of research at Parent Project Muscular Dystrophy (PPMD), the largest DMD non-profit in the U.S. In two meetings already held with the FDA’s neurology division, Hesterlee says PPMD made sure the agency understands that “time is extremely critical” for DMD patients because the disease is relentlessly progressive. Every week and month that goes by means DMD patients lose more muscle function. Without treatment, DMD patients end up in wheelchairs and often die in their 30s.
