The FDA has approved Emflaza (deflazacort) tablets and oral suspension for the treatment of patients aged 5 years and older with Duchenne muscular dystrophy.
Emflaza, a corticosteroid, works by decreasing inflammation and reducing activity of the immune system.
The drug was approved based upon a clinical study of 196 male patients aged 5 to 15 years old at the beginning of the trial. At week 12, those taking deflazacort showed improvements in a clinical assessment of muscle strength compared with placebo. Overall stability in average muscle strength was maintained through week 52 in the deflazacort group. In a second trial of 29 male patients, deflazacort was associated with a numerical advantage over placebo on an assessment of average muscle strength.
Common adverse effects included facial puffiness, weight gain, increased appetite, upper respiratory tract infection, cough, unwanted hair growth, and central obesity.
—Michael Potts
Reference:
US Food and Drug Administration. FDA approves drug to treat Duchenne muscular dystrophy [press release]. February 9, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm540945.htm.
