FDA Approves Duchenne Muscular Dystrophy Drug

Emflaza duchenne muscular dystrophy
Emflaza duchenne muscular dystrophy
Emflaza is approved for the treatment of Duchenne muscular dystrophy regardless of genetic etiology.

The Food and Drug Administration (FDA) has approved Emflaza (deflazacort; Marathon) tablets and oral suspension for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 5 years and older. 

The FDA approval makes Emflaza the first corticosteroid to treat DMD. DMD is a rare, genetic disease caused by mutations in the dystrophen gene. It primarily affects boys, occurring in about 1 out of every 3600 male infants worldwide. Deflazacort works by reducing inflammation and suppressing the immune system, however the exact mechanism by which the drug exerts its therapeutic effects in patients with DMD is unknown.

The efficacy of Emflaza was demonstrated in a clinical study (N=196) of male patients aged 5 to 15 years at the start of the trial with documented mutation of the dystrophin gene and onset of weakness before age 5. At week 12,  patients in the Emflaza group had improvements in clinical assessment of muscle strength across a number of muscles vs patients taking placebo. By week 52, patients in the Emflaza group maintained an overall stability in average muscle strength. 

In another trial (N=29) of male patients lasting 104 weeks, treatment with Emflaza had a numerical advantage over placebo on an assessment of average muscle strength. Patients in the Emflaza arm seemed to lose their walking ability later than patients in the placebo arm, though this was not statistically controlled for multiple comparisons.

The most common adverse effects with Emflaza included Cushingoid appearance, weight gain, increased appetite, upper respiratory tract infection, cough, pollakiuria, hirsutism, and central obesity. 

Emflaza will be available as 6mg strength tablets in 100-count bottles and as 18mg, 30mg, and 36mg strength tablets in 30-count bottles. It will also be supplied as a 22.75mg/mL strength oral suspension in 20mL bottles with oral dispensers. 


FDA approves drug to treat Duchenne muscular dystrophy [news release]. US FDA newsroom; February 9, 2017. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm540945.htm. Accessed February 13, 2017. 

This article originally appeared on MPR