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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.

Citation:

US Food and Drug Administration. FDA approves drug to treat Duchenne muscular dystrophy. FDA Web site. February 9, 2017. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm540945.htm. Accessed February 13, 2017.