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FDA approves Marathon Pharmaceuticals’s orphan drug

The U.S. Food and Drug Administration (FDA) has approved Marathon Pharmaceuticals’s Emflaza (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.

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

Even though corticosteroids are commonly used to treat DMD worldwide, this is the first FDA approval of corticosteroid to treat DMD and the first approval of deflazacort for any use in the United States.

DMD is the most common type of muscular dystrophy that occurs in about one of every 3,600 male infants worldwide. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact.

 

 

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