In a Thursday press release, the Food and Drug Administration (FDA) granted approval for a drug by Marathon Pharmaceuticals, a US company focused solely on the development of new treatments for rare diseases. They may now begin selling the corticosteroid drug Deflazacort, under the brand name Emflaza.
With FDA approval, Marathon now has exclusive rights to sell the drug in the US for the next seven years, even though it has been available as a generic in other countries.
With exclusive rights, the company can increase the price from the $1,200 average that families were importing the drug from overseas to $89,000, an increase of more than 7,000 percent.
Deflazacort is used to treat Duchenne muscular dystrophy (DMD), a rare genetic disease that causes heart and respiratory conditions, usually in younger men. As the disease progresses, patients lose muscle strength, and usually end up confined to a wheelchair by the time they reach their early teens.
Patients with DMD typically do not live past their 20s or 30s, according to the FDA.
“This is the first treatment approved for a wide range of patients with Duchenne muscular dystrophy,” Billy Dunn, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research, said in the press release. “We hope that this treatment option will benefit many patients with DMD.”
Since so few Americans are affected by the disease, only around 15,000, the drug was approved under an “orphan drug” status, which is a special status given to drugs that treat rare diseases or conditions.
Although the price has increased, Babar Ghias, Marathon’s Chief Financial Officer, says that drug is still in the bottom 10 percentile of rare disease drug prices.
Ghias also says that that after rebates and discounts, the price will drop to $54,000, and with insurance and financial programs most patients will pay “zero to low out-of-pocket expense,” according to the Washington Post.