Drug Therapy for Duchenne MD Seeks Fast-Track Approval from FDA

A drug commonly used in other countries to help treat Duchenne muscular dystrophy may soon become available in the United States. Deflazacort has been available for decades in the UK, Canada, and Mexico, but many Americans have had to rely on overseas shipment of the drug to help treat their child’s muscular dystrophy as it is not currently approved for use in America. Learn more about this promising development in today’s blog.

A new drug seeking approval could help those living with Duchenne MD.

Drug Therapy for Duchenne MD Seeks Fast-Track Approval from FDA

What is Duchenne muscular dystrophy?

Duchenne muscular dystrophy currently affects between 15,000-20,000 boys in the US. Though women may be carriers of the disease, Duchenne almost always shows symptoms in boys who are generally between the ages of 2-5. The disease is degenerative and causes significant muscle weakness but with treatment, many patients can live into their 40s.

What is deflazacort?

While it cannot cure Duchenne MD, deflazacort has been shown to extend lives by helping patients continue to walk and breathe. A steroid that is related to prednisone, deflazacort is used for anti-inflammatory purposes and also acts as an immunosuppressant. Studies have shown that patients with muscular dystrophy who receive the drug experience less bone density loss and are less likely to develop scoliosis.

When will the FDA approve the drug?

Despite decades of use in other countries, the company that developed deflazacort never sought approval in the US. While its use is legal because no other treatments for Duchenne are available, the drug cannot be obtained domestically and must be shipped from overseas which currently costs around $125/month.

Because of the drug’s long track record, the fact that the affected population is fewer than 200,000 people, and no other treatment is currently available, the FDA has agreed to fast-track its application status. Marathon Pharmaceuticals, the company hoping to bring the drug to the US, is currently conducting clinical trials with the hope that the drug will be approved by the end of 2016.


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