The U.S. Food and Drug Administration (FDA) has approved a drug meant to treat Duchenne Muscular Dystrophy. The drug, Emflaza (deflazacort), is available in both tablet and liquid form and can be used by patients ages five years and older.
Duchenne Muscular Dystrophy is a rare genetic disorder that causes progressive muscle deterioration and weakness. Symptoms of the disorder typically appear between between the ages of 3 and 5—affecting the patient for years to come. It is common for patients to die in their 20s or 30s.
Emflaza is a corticosteroid that works to decrease inflammation and reduce the activity of the immune system, which thus improves muscle strength in patients. Though corticosteroids are used to treat Duchenne Muscular Dystrophy in foreign countries, the FDA was hesitant to approve the drug prior to extensive research and testing.
The FDA’s ultimate approval of emflaza was based on two studies showing that male patients who took the drug had improved muscle strength, giving them more years with the ability to walk. In one study, 196 male patients between the ages of five and 15 were tested. After 12 weeks, there were improvements, and muscles appeared to be stronger in patients that took the drug. In the second study, looked at 29 male patients over 104 weeks, and showed similar results.
Though there are some side effects associated with the drug, its potential outweighs these minor negative aspects.
“This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy,” said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “We hope that this treatment option will benefit many patients with DMD.”
Photo: David K, CC-BY
Elizabeth Chambers is a health intern with Paste and a freelance writer based out of Athens, Georgia.