Marathon Pharmaceuticals’ Duchenne muscular dystrophy drug wins FDA approval

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

The drug’s effectiveness was demonstrated in a clinical study of 196 boys with DMD aged 5 to 15 years.

At week 12, patients receiving deflazacort had improvements in muscle strength compared with those taking a placebo, which was maintained at week 52.

In a separate trial of 29 male patients lasting 104 weeks, deflazacort showed a numeric advantage over placebo on an assessment of average muscle strength.

Patients receiving deflazacort also appeared to lose the ability to walk later compared to those treated with placebo.

Adverse effects related to deflazacort mirror were same to those seen with other corticosteroids, the FDA said.

The most common side effects include facial puffiness (cushingoid appearance), weight gain, increased appetite, upper respiratory tract infection, cough, pollakiuria, hirsutism, and central obesity.

Less common side effects include problems with endocrine function, increased susceptibility to infection, elevation in blood pressure, risk for gastrointestinal perforation, serious skin rashes, behavioral and mood changes, decrease in the density of the bones, and vision problems.

Marathon Pharmaceuticals executive vice president of research & development Timothy Cunniff said: “For the first time, patients in the U.S. with Duchenne will have widespread access to an FDA approved medicine that is indicated for all genetic forms of the condition.

“This is only the first of our products to treat Duchenne muscular dystrophy and we are committed to serving this community of patients, researchers and advocates until a cure is found.”

Image: FDA approves Emflaza tablets and oral suspension to treat Duchenne muscular dystrophy in patients 5 years and older. Photo: courtesy of Business Wire.