CHICAGO _ The Food and Drug Administration has agreed to consider green-lighting a drug that could be among the first of its kind approved to treat a childhood-onset form of muscular dystrophy, Marathon Pharmaceuticals announced Wednesday.
Marathon, headquartered in suburban Chicago, has the U.S. rights to the drug, called deflazacort. The pharmaceutical is already available and used to treat Duchenne muscular dystrophy outside the U.S., where it's approved for other uses. But because the steroid does not have FDA approval, it's not widely available here.
"This would be a big step forward for families across the country to have access to the drug in the U.S.," said Valerie Cwik, Muscular Dystrophy Association executive vice president and chief medical and scientific officer.
Duchenne muscular dystrophy is a genetic disorder that mostly affects boys. It can cause movement, heart and breathing problems and typically leads to death in early adulthood, Cwik said. It's the most common form of childhood-onset muscular dystrophy, she said.
Now, those with Duchenne are often treated with prednisone, also a steroid, though it's not specifically approved to treat the disorder.
Prednisone, however, can cause more weight gain as a side effect than deflazacort, Cwik said.
The FDA agreed to give the deflazacort drug applications _ one for tablet and one for a liquid _ priority review status, meaning it could have a decision in six months rather than 10. A decision is expected in February, according to Marathon.
One other drug meant to treat the disease, eteplirsen, developed by Sarepta Therapeutics, is also under FDA review.
During the FDA review process Marathon is offering the drug for free to patients in the U.S. who work with participating doctors through its FDA-authorized Access DMD program, said Tim Cunniff, Marathon's executive vice president of research and development. To learn more visit www.AccessDMD.com or call 844-800-4363.
