FDA approves muscular-dystrophy drug costing $89,000 in the US

A drug that had been on sale to treat a rare form of muscular dystrophy has been approved by the FDA for sale in the US, but it will come at a high price.

According to The Wall Street Journal, the FDA-approved deflazacort would be offered by Marathon Pharmaceuticals for $89,000 or so a year, up to 70 times its cost outside the US.

However Marathon CFO Babar Ghias told The Washington Post that the net price would be $54,000 after rebates and discounts.

The high price had led to dismay among patyients of Duchenne muscular dystrophy, the health condition it is expected to treat. They fear they would not be able to afford the treatment, if insurance would not pay for it.

Ghias told the Journal that the drug which would be sold under the brand Emflaza starting in March.

Meanwhile, the FDA said in a press release, "The US Food and Drug Administration today approved Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 years and older with Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness. Emflaza is a corticosteroid that works by decreasing inflammation and reducing the activity of the immune system."

Corticosteroids are commonly used to treat DMD across the world. This is the first FDA approval of any corticosteroid to treat DMD and the first approval of deflazacort for any use in the United States.

''This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy,'' said Billy Dunn, MD, director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research. ''We hope that this treatment option will benefit many patients with DMD.''

DMD is the most common type of muscular dystrophy, and is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. The first symptoms are usually seen between 3 and 5 years of age and worsen over time.

The disease often occurs in people without a known family history of the condition and primarily affects boys, but in rare cases it can affect girls. DMD occurs in about one of every 3,600 male infants worldwide.