King, Colleagues Demand Answers on High Price of Drug for Patients with Rare Genetic Disorder

Press Release

U.S. Senator Angus King (I-Maine) announced today that he has joined Senator Patty Murray (D-Wash.), the ranking member of the Senate Health, Education, Labor and Pensions (HELP) Committee, and six of their colleagues in sending a letter to the Chairman and CEO of Marathon Pharmaceuticals, Jeffrey S. Aronin, demanding answers regarding the initial proposed pricing scheme for Marathon Pharmaceuticals' recently approved steroid for use in patients with Duchenne Muscular Dystrophy (DMD). Marathon intended to sell its treatment, Emflaza (deflazacort), for $89,000, despite the fact that deflazacort is not a new innovation for DMD.

"The circumstances surrounding the development of Emflaza, and the benefits that will accrue to Marathon as a result of its approval as a new drug, raise serious questions about whether there is any justification for such a dramatically high price," Senator King and his colleagues wrote. "We are concerned that Marathon's pricing unfairly exploits the DMD patient population and the FDA's orphan drug incentives."

While treatments for "orphan diseases" -- those that affect less than 200,000 patients nationwide -- are often more expensive than drugs for diseases that impact more patients, deflazacort has been used to treat DMD for decades. In the letter, Senator King and his colleagues sent a series of questions about Marathon's pricing scheme, including how much Marathon has and anticipates investing on research and development of deflazacort, and how much revenue the company anticipates from marketing deflazacort during its seven year monopoly.

Along with Senators King and Murray, the letter was signed by Senators: Tammy Baldwin (D-Wisc.), Cory A. Booker (D-N.J.), Al Franken (D-Minn.), Maggie Hassan (D-N.H.), Elizabeth Warren (D-Mass.), and Sheldon Whitehouse (D-R.I.).

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