A New Rx for Duchenne Muscular Dystrophy

The US Food and Drug Administration (FDA) has approved a new medication for the most common type of muscular dystrophy.

It's called Emflaza (deflazacort), and it was approved to treat patients ages 5 and older who have Duchenne muscular dystrophy.

This type of muscular dystrophy is considered the most common. It's a rare, progressive genetic disorder that causes weakness and muscle deterioration. The symptoms tied to this condition usually start to show between the ages of 3 and 5.

Emflaza, which is available in tablet and oral suspension forms, belongs to a group of drugs called corticosteroids.

"This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy," said Dr. Billy Dunn, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, in a press release.

This approval comes after a clinical study of more than 190 male participants between the ages of 5 and 15. After 12 weeks, those who received deflazacort saw an improvement in muscle strength compared to those taking a placebo.

Common side effects included facial puffiness, weight gain, increased appetite, upper respiratory tract infection, unwanted hair growth and cough. 

Marathon Pharmaceuticals markets Emflaza.