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FDA approves first treatment for Duchenne muscular dystrophy

Published on 10/02/17 at 09:06am

The FDA has announced that it has approved Emflaza (deflazacort), developed by Marathon Pharmaceuticals. The treatment will arrive on the market as the first treatment for Duchenne muscular dystrophy, a muscle-wasting disease that primarily affects young boys. The drug has been granted approval to treat children above the age of five.

Ostensibly, this appears to be good news but there is a caveat. Previously, patients were able to import the drug at a low cost because there was no designated treatment available within the US. Now, due to the approval, patients will not be able to do this and this could see them pay more for the drug.

Marathon has stated that the list price will be $89,000 a year for the drug - by most measures, this can only be regarded as an expensive treatment. However, Marathon has insisted that patients who are not covered by insurance will be able to receive assistance programs to help them receive the drug.

“We are in a new era in the treatment of Duchenne muscular dystrophy. 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. We are pleased that this development will help patients with this disease stay stronger longer,” said Timothy M. Cunniff, Executive Vice President, Research & Development, Marathon Pharmaceuticals. “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.”

In line with Cunniff’s comments, Marathon plan to conduct further post-market research looking into further dosage regimens, including within younger age groups than the current approval.

The research that gained the FDA’s approval was focused on 196 boys between the ages of 5 and 15 years of age. The 52-week study found that 12-weeks of treatment resulted in continued benefits in muscle strength up until the end of the study.

Ben Hargreaves

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