Lawmakers question pricing of drug for genetic disease

WASHINGTON >> Marathon Pharmaceuticals’ pricing of a drug to treat genetic muscle deterioration in about 15,000 Americans, mostly boys, is raising concerns in Congress where lawmakers repeatedly have challenged drug companies.

Sen. Bernie Sanders, I-Vt., and Rep. Elijah Cummings, D-Md., complained on Monday that Marathon Pharmaceuticals plans to charge $89,000 a year for a drug that’s widely available abroad for about $1,000 a year.

The two lawmakers said they believe Marathon is abusing a program that grants companies seven years of market exclusivity to encourage research into new treatments for rare diseases. They said the program was not intended to provide companies lucrative exclusivity rights for drugs that have been available for decades.

“We urge you to significantly lower your price for this drug before it goes on the market next month,” the lawmakers wrote.

The U.S. Food and Drug Administration approved the drug last week to treat patients with Duchenne Muscular Dystrophy. While the steroid is commonly used globally, the FDA’s action represents the first approval of Deflazacort for use in the U.S. The drug will be marketed under the brand name Emflaza.

The two lawmakers say that “exorbitantly” pricing potentially life-saving medications hurts patient access and drives up prices for the entire health care sector. They are investigating how Marathon set its pricing and want to know the company’s total expenses related to development and approval of the drug.