FDA Approves Corticosteroid Treatment for Duchenne Muscular Dystrophy

FDA officials today announced the approval of Emflaza (deflazacort) tablets and oral suspension for the treatment of Duchenne muscular dystrophy (DMD). The drug is indicated to treat patients aged 5 years and older with the rare genetic disorder.

Emflaza, which is marketed by Marathon Pharmaceuticals, is a corticosteroid that decreases inflammation and reduces the activity of the immune system, thereby relieving the effects of progressive muscle deterioration and weakness. Although corticosteroids are commonly used to treat DMD, this is the first FDA-approved corticosteroid treatment and the first approval of deflazacort in the United States.

The approval was based on a clinical study of 196 male participants that demonstrated the effectiveness of deflazacort in patients with documented mutation of the dystrophin gene and onset of weakness before age 5. The study researchers noted an overall improvement in muscle strength, and patients taking deflazacort appeared to lose the ability to walk later than those who took a placebo.

“This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy,” Billy Dunn, MD, director of the Division of Neurology Products in the FDA’ Center for Drug Evaluation and Research, said in a press release. “We hope that this treatment option will benefit many patients with DMD.”

Adverse effects associated with Emflaza include facial puffiness, weight gain, increased appetite, upper respiratory tract infection, cough, extraordinary daytime urinary frequency, unwanted hair growth, and excessive fat around the stomach.

Reference

FDA approves drug to treat Duchenne muscular dystrophy [news release]. FDA’s website. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm540945.htm. Feb. 9, 2017.