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FDA Approves Drug to Treat Duchenne MD

FDA news release; 2017 Feb 9

The US Food and Drug Administration (FDA) has approved Emflaza (deflazacort) tablets and oral suspension to treat patients aged ≥5 years with Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness. Emflaza is marketed by Marathon Pharmaceuticals of Northbrook, IL.

Indications: Emflaza is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system.

Dosage/administration: Emflaza is administered orally by tablet; it is available in 6 mg, 18 mg, 30 mg, and 36 mg (oral suspension: 22.75 mg/mL). The recommended once-daily dosage is approximately 0.9 mg/kg/day. Discontinue gradually when administered for more than a few days.

Adverse effects: The most common adverse reactions (≥ 10% for Emflaza and greater than placebo) are Cushingoid appearance, weight increase, increased appetite, upper respiratory tract infection, cough, pollakiuria, hirsutism, central obesity, and nasopharyngitis.


US Food and Drug Administration. FDA approves drug to treat Duchenne muscular dystrophy. FDA Web site. February 9, 2017. Accessed February 13, 2017.