) -- Duchenne muscular dystrophy (DMD) advocacy groups are mobilizing to support efforts by
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.
"We made the point to FDA that we already know the risk of doing nothing in DMD. The risk tolerance for DMD patients is very high," says Hesterlee.
The Duchenne Alliance, an umbrella advocacy group that represents the interests of 37 smaller DMD charities and foundations worldwide,
submitted a dossier to FDA
last week to get DMD included on a list of rare diseases eligible for special considerations, including accelerated access and approval, under the recently approved "PDUFA V" legislation.
"We have such stellar, unprecedented data with eteplirsen. We really need to carry the flag on this," said Christine McSherry, Duchenne Alliance's executive director and the mother of a son with DMD.
After 48 weeks of treatment with eteplirsen at the highest 50 mg dose, four boys with DMD were able to walk 21 meters further than they could when the study started. Another four boys who started the study on placebo but then switched to eteplirsen after 24 weeks saw a stabilization of their walking ability, with six-minute walk test performance improving to a 68-meter decline at 48 weeks from a 78-meter decline at 36 weeks.