Recently, U.S. Food and Drug Administration today approved deflazacort tablets and oral suspension to treat patients age 5 years and older with Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness. Deflazacort is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system.
This is the first FDA approval of any corticosteroid to treat DMD. This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy.
DMD is the most common type of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. The first symptoms are usually seen between 3 and 5 years of age and worsen over time. The disease often occurs in people without a known family history of the condition and primarily affects boys, but in rare cases it can affect girls. DMD occurs in about one of every 3,600 male infants worldwide.