The US Food and Drug Administration today approved Emflaza (deflazacort) tablets and oral suspension to treat patients aged five years and older with Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness.

Emflaza is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system.

Corticosteroids are commonly used to treat DMD across the world. This is the first FDA approval of any corticosteroid to treat DMD and the first approval of deflazacort for any use in America.

‘This is the first treatment approved for a wide range of patients with DMD,’ commented Billy Dunn, MD, Director of the Division of Neurology Products in the FDA’s Centre for Drug Evaluation and Research.

‘We hope that this treatment option will benefit many patients with DMD.’

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 three and five 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.

People with DMD progressively lose the ability to perform activities independently and often require use of a wheelchair by their early teens. As the disease progresses, life-threatening heart and respiratory conditions can occur. Patients typically succumb to the disease in their 20s or 30s; however, disease severity and life expectancy vary.

Emflaza is marketed by Marathon Pharmaceuticals of Northbrook, Illinois.