sNDA for Emflaza Expands Patient Population

FDA has approved a PTC Therapeutics supplemental NDA for Emflaza (deflazacort) to expand its labeling to include patients with Duchenne muscular dystrophy (DMD) who are between two and five years of age. Emflaza was first approved 2/2017 for treating Duchenne in patients five years and older. An agency news release says that Emflaza is a corticosteroid that works by decreasing inflammation and reducing immune system activity. Although corticosteroids are commonly used to treat DMD across the world, this was the first FDA approval of any corticosteroid to treat DMD. Side effects caused by Emflaza are similar to those experienced with other corticosteroids, the agency says. The most common include facial puffiness, weight gain, increased appetite, upper respiratory tract infection, cough, extraordinary daytime urinary tract frequency, unwanted hair growth, and excessive fat around the stomach.