FDA approves new drug to treat Duchenne muscular dystrophy

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

Emflaza, which will be marketed by privately-held Marathon Pharmaceuticals, 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, but this is the first FDA approval of any corticosteroid to treat DMD and the first approval of deflazacort for any use in the USA.

“This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy,” said Billy Dunn, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, adding: “We hope that this treatment option will benefit many patients with DMD.”