FDA Approves Emflaza for Duchenne Muscular Dystrophy
Approved to treat Duchenne muscular dystrophy in patients 5 years and older
FRIDAY, Feb. 10, 2017 (HealthDay News) -- Emflaza (deflazacort) has been approved by the U.S. Food and Drug Administration to treat Duchenne muscular dystrophy (DMD) in patients 5 years and older, the agency said Thursday in a news release.
The corticosteroid is designed to reduce inflammation and suppress the immune system in patients with DMD, the most common type of muscular dystrophy. The FDA said it's the first corticosteroid approved to treat DMD.
Emflaza was evaluated in a clinical study of 196 males aged 5 to 15, all with documented mutation of the dystrophin gene and onset of DMD symptoms before age 5. After 12 weeks, patients taking the drug showed muscle strength improvement, compared with those who took a placebo.
Side effects, similar to those caused by other corticosteroid medications, included facial puffiness, weight gain, increased appetite, upper respiratory tract infection, cough, and increased need to urinate, the FDA said.
Emflaza is marketed by Marathon Pharmaceuticals, based in Northbrook, Ill.