The drug has been approved for treatment of Duchenne muscular dystrophy in patients 2– 5 years of age.
Emflaza (deflazacort) tablets and oral suspension from PTC Therapeutics were approved in February 2017 by the U.S. Food and Drug Administration (FDA) for treatment of patients 5 years and older with Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness.
DMD largely affects boys and leads to premature death in the mid-twenties due to heart and respiratory failure. Emflaza is a corticosteroid that decreases inflammation and reduces immune system activity.
The FDA has now allowed use of Emflaza for the treatment of children 2–5 years old with the approval of PTC’s supplemental New Drug Application (sNDA). According to PTC Therapeutics CEO Stuart Peltz, treatment with Emflaza of younger patients who still have significant muscle mass should provide the greatest benefit.