FDA approved Marathon Therapeutics’s oral corticosteroid drug Emflaza™ (deflazacort) for treating Duchenne muscular dystrophy (DMD) in patients aged 5 years and over, irrespective of the underlying genetic mutation. FDA is the first regulator to approve the drug for treating all genetic forms of the disorder. 

“This is only the first of our products to treat DMD, and we are committed to serving this community of patients, researchers, and advocates until a cure is found,” commented Timothy M. Cunniff, Pharm.D., evp R&D at Marathon.” For the first time, patients in the U.S. with Duchenne will have widespread access to a FDA-approved medicine that is indicated for all genetic forms of the condition.'

“Emflaza is an important new drug with proven benefit in boys with Duchenne muscular dystrophy, increasing muscle strength and physical function, which is important for both patients and caregivers,” added Robert C. Griggs, M.D., University of Rochester Medical Center, an early investigator of deflazacort. “In my own research and in the clinic, deflazacort keeps boys walking longer. By undertaking the research needed to secure FDA approval of Emflaza, we now know more about the drug, its dosing, and possible interactions.”

Clearance of Emflaza followed priority review by FDA. A 52-week, placebo-controlled study of deflazacort in 196 male patients aged 5–15 years showed that treatment with the drug led to significant improvements in muscle strength throughout the study duration. Marathon will carry out a number of post-marketing Emflaza studies to investigate potential dosage regimens in younger patients and in nonambulatory patients and to characterize pulmonary and cardiac effects of the drug.

Marathon is focused on developing treatments for complex rare diseases, including pediatric juvenile idiopathic arthritis, neuromyelitis optica, idiopathic inflammatory myopathies, catastrophic pediatric epilepsy, and multiple sclerosis. The firm’s clinical pipeline includes MP-102, which is in late-stage development for a pediatric neurology indication.

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