The Food and Drug Administration (FDA) has approved an expanded indication for Emflaza (deflazacort; PTC Therapeutics, Inc.) to include children 2 to 5 years of age with Duchenne muscular dystrophy (DMD).

Emflaza was initially approved in February 2017 for the treatment of DMD in patients 5 years and older. Approval for patients 2 years to <5 years of age was supported by data from a 52-week phase 3 double-blind, placebo-controlled study of 196 patients aged 5 to 15 years with DMD.

The prescribing information has also been updated to include additional information regarding immunizations. Immunizations should be administered according to guidelines prior to starting Emflaza. Patients on Emflaza may receive concurrent vaccinations, except for live-attenuated or live vaccines; these should be administered at least 4 to 6 weeks prior to starting treatment.

“The standard of care is to start Emflaza at the time of diagnosis,” said Stuart Peltz, PhD, Chief Executive Officer of PTC Therapeutics. “We believe that treating patients as young as possible, when they still have a substantial amount of muscle, will have the greatest benefit for patients that are 2 years and older.”

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PTC Therapeutics plans to initiate a phase 3 study of Emflaza in patients with limb-girdle muscular dystrophy with an estimated completion date of August 2020.

Emflaza is currently the only approved treatment for patients 2 years of age and older with DMD. It is available as a tablet or oral suspension.

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