FDA grants rare pediatric disease status to deflazacort for Duchenne's muscular dystrophy

The FDA has issued rare pediatric disease designation to deflazacort for the treatment of patients with Duchenne’s muscular dystrophy, according to the drug’s manufacturer.

A genetic disorder characterized by progressive muscle degeneration, difficulty walking and breathing, and eventually death, Duchenne’s muscular dystrophy is typically diagnosed in children aged 2 to 5 years with progressive weakness and loss of ambulation in adolescence.

Although Duchenne’s muscular dystrophy affects approximately 15,000 boys in the United States, there are no currently approved therapies for this disease, the release said.

An oxazoline derivative of prednisolone, deflazacort (Marathon Pharmaceuticals) is a glucocorticoid prescribed for anti-inflammatory conditions and used as an immunosuppressant. While not approved in the U.S., deflazacort has been widely used in Canada and Europe.

The FDA based its decision on previously published clinical studies among children with Duchenne’s muscular dystrophy. Those studies found that — compared with prednisone — deflazacort better preserved bone density with long-term use; additionally, patients were less likely to develop scoliosis. Adverse events reported in clinical trials included cushingoid appearance, hirsutism, weight gain, erythema, nasopharyngitis, irritability and cataracts.

This rare pediatric disease designation supplements the orphan drug designation granted by the FDA in August 2013 for deflazacort for the treatment of patients with Duchenne’s muscular dystrophy. According to a press release, the company expects to submit a new drug application for deflazacort to the FDA in the first half of 2016.