What type of drug is Emflaza, how does it work?

Key Points

• Emflaza (deflazacort) is a glucocorticoid (a corticosteroid or "steroid") used to treat Duchenne Muscular Dystrophy (DMD) in patients age 2 years of age and older.
• Emflaza is not a cure for muscular dystrophy, but it may improve a patient's muscle strength and help to slow the progression of the disease.
• Emflaza is the first FDA approval of any corticosteroid to treat DMD in the U.S. It was approved by the FDA in 2017. It comes as an oral suspension or as an oral tablet taken by mouth once a day. Doses are based on the patient's weight.

How does Emflaza work in DMD?

Duchenne muscular dystrophy (DMD) is a genetic disorder that most common occurs in boys. It is rarely found in girls. DMD affects muscles in the body and leads to weakness. It usually starts in the lower limbs and pelvis areas. There is no cure and it often leads to premature death in the mid-twenties to thirties due to heart and lung failure.

Corticosteroids in general help to reduce inflammation (swelling) and pain. They can help to improve motor skills, muscle strength, lung function, and delay walking disability. Emflaza is manufactured by PTC Therapeutics.

Studies have shown that beginning treatment early in Duchenne Muscular Dystrophy (DMD) before physical decline is important for treatment. The U.S. Centers for Disease Control and Prevention (CDC) recommends that boys with DMD begin corticosteroid therapy before they show substantial physical decline. The American Academy of Neurology (AMA) also recommends corticosteroid use as part of Duchenne treatment.

Other treatments used for the treatment of DMD include devices like braces or crutches, counseling, physical and occupational rehabilitation, and sometimes surgery to help with severe muscle tightening or shortening (contracture).

Learn more: Emflaza side effects (in detail)

This is not all the information you need to know about Emflaza (deflazacort) for safe and effective use and does not take the place of talking to your doctor about your treatment. Review the full Emflaza information here, and discuss this information and any questions you have with your doctor or other health care provider.