New price for muscular dystrophy drug draws criticism | CNBC

Deflazacort wasn’t approved in the U.S., so McSherry and a number of other families of kids with Duchenne imported the drug from overseas. She says it cost about $1,200 a year out of pocket.

Monday, deflazacort got a new price tag: $35,000 a year for a patient in the U.S. weighing 25 kilograms, or about 55 pounds.

To McSherry, the $35,000 is “pretty aggressive.” What’s worse, in her eyes, is that most kids who may take the drug weigh more than 25 kilograms, meaning the price will be even higher.

“You have young boys, as little as 30 kilograms, to young men who are 100-plus kilograms,” McSherry said. “That price is up there over $100,000 annually.”

PTC Therapeutics, which set the price, says it “represents sustainable pricing which balances providing access to eligible patients in the United States…while maintaining sufficient infrastructure and programs including continued investment in Duchenne.”

The route to deflazacort’s new price is a complicated one. Earlier this year, a private drug company called Marathon Pharmaceuticals acquired rights to deflazacort in the U.S. and took it through the approval process at the Food and Drug Administration. Upon approval, Marathon set an annual price in the U.S. of $89,000, generating an immediate outcry from the patient community. Once the medicine got approval in the U.S., patients expected they wouldn’t be able to legally import it from overseas.

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New price for muscular dystrophy drug draws criticism.