New Drug Approved For Muscular Dystrophy

On Thursday, the Food and Drug Administration (FDA) approved the drug Emflaza (deflazacort) for the treatment of Duchenne Muscular Dystrophy (DMD). The drug will be available in both oral suspension and tablets, and can be administered to patients 5 years of age and older.

DMD is a rare genetic muscle disorder that is characterized by progressive muscle deterioration and weakness. The disorder is caused when there is an absence of the protein dystrophin, which helps to keep the muscles intact, according to the FDA.

DMD typically affects boys; however, girls can be mildly affected and be carriers of the gene, according to the Muscular Dystrophy Association. There is no cure for DMD, but medications can help improve a patient’s quality of life. Symptoms include falls, difficulty getting up from a seated position, difficulty running, a waddling gait, large calves, and difficulty learning.

People who have DMD eventually lose the ability to carry out activities on their own and are wheelchair bound by the time they reach their early teen years. As the disease continues to progress, patients can develop life threatening respiratory and heart related conditions. While life expectancy varies per patient due to the severity of their individual case of DMD, patients typically pass away in their 20s or 30s

Emflaza is a corticosteroid that treats the disorder by reducing activity within the immune system and decreasing inflammation. This is the first corticosteroid approved by the FDA to treat patients who have Duchenne muscular dystrophy. Dr. Billy Dunn of the Division of Neurology Products with the Center for Drug Evaluation and Research at the Food and Drug Administration said the FDA is hopeful that Emflaza will help a wide range of patients with DMD.

Side effects associated with taking Emflaza are similar to that of other corticosteroids including, puffiness of the face, weight gain, an increased appetite, infection of the upper respiratory tract, frequent urination, cough, stomach fat, and unwanted hair growth. Other side effects include elevated blood pressure, mood changes, infection, and serious skin rashes.

Source: CDA news