New Muscular Dystrophy Drug to Cost $89K a Year

Feb. 13, 2017 -- A steroid drug long available in other countries recently received U.S. Food and Drug Administration approval to treat a rare disease called Duchenne muscular dystrophy, which affects about 15,000 Americans.

U.S. patients and their families have long been able to import deflazacort for about $1,200 a year on average, but the new list price for the drug in the U.S. is $89,000, the Washington Post reported.

After rebates and discounts, the net price of the drug that goes by the brand name Emflaza will be $54,000 a year, according to Babar Ghias, chief financial officer of Marathon Pharmaceuticals.

The cost of the drug for patients would be "zero to low out-of-pocket expense," because of insurance and financial assistance programs that the company will offer, Ghias told the Post.

Duchenne muscular dystrophy is a genetic disease that robs boys of the ability to walk by the time they're in their teens and kills them as young adults. Studies show that deflazacourt helps patients retain muscle strength, the Post reported.

The FDA's approval of Emflaza was praised by the patient advocacy group Parent Project Muscular Dystrophy. In a statement, the group said it hopes "this means easier access for more families. There are many questions still to be answered, including pricing, and we look forward to talking to Marathon with the community to begin to answer those questions."

University of California at Davis Professor Craig McDonald told the Post he has used deflazacort in his clinic for a decade and that about 60 of his patients with Duchenne muscular dystrophy are taking the drug.

Most of his patients receive the drug for free through an expanded access program funded by Marathon. Many patients he sees -- whose parents are migrant workers or covered by California's Medicaid program -- can't afford $1,200 out of pocket to import the drug.

"Now the hope is at least all children will have access to an actual FDA approved therapy for Duchenne," McDonald told the Post.

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