FDA approves corticosteroid for muscular dystrophy

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Food and Drug Administration granted its first approval of a corticosteroid to treat Duchenne muscular dystrophy (DMD).

Deflazacort, which is being marketed as Emflaza by Marathon Pharmaceuticals, is meant to treat patients who have DMD and are age 5 and older by decreasing muscle inflammation and reducing the activity of the immune system. Corticosteroids, such as deflazacort, are used commonly to treat DMD globally and many illnesses here.

DMD is characterized by progressive muscle deterioration and weakness, and it’s the most common form of muscular dystrophy. DMD is caused by an absence of dystrophin, which is a protein that helps to keep muscle cells intact. The first symptoms typically are seen in patients who are ages 3–5, and they get worse over time. Patients progressively lose the ability to perform activities independently and typically don’t live beyond their 30s. The disease often occurs in people who don’t have a family history of it, and it affects boys primarily. DMD occurs in about 1 of every 3,600 male infants worldwide.

In a clinical study of 196 patients, deflazacort was shown to improve muscle strength across a number of muscles compared with a placebo after 12 weeks.

Potential side effects for deflazacort are similar to those that other corticosteroids have, FDA says. Those include facial puffiness, weight gain, increased appetite, upper respiratory tract infection, cough, extraordinary daytime urinary frequency, unwanted hair growth and excessive fat around the stomach. Less common potential side effects include increased susceptibility to infection, elevated blood pressure, risk of gastrointestinal perforation, serious skin rashes, behavioral or mood changes, decreased bone density and vision problems such as cataracts. Patients who receive immunosuppressive doses of corticosteroids shouldn’t receive live or live attenuated vaccines, FDA says.