An old drug gets new price and congressional investigation

Marathon Pharmaceuticals has delayed plans to market an old drug under a new label after members of Congress expressed outrage that the company would charge U.S. consumers $89,000 for the drug, compared to a $2,000 outside the United States.

The U.S. Food and Drug Administration recently approved deflazacort to treat patients with Duchenne Muscular Dystrophy. About 15,000 Americans, mostly boys, suffer from the rare and deadly disease.

While deflazacort, a steroid, is not new, Marathon Pharmaceuticals has successfully applied for and received “orphan drug” designation from the FDA.

The “orphan drug” program was designed to encourage drug makers to develop medications to treat rare diseases. It also gives drug makers seven years of exclusive right to sell the drug in the United States.

Critics have accused the pharmaceutical industry of using the “orphan drug” program to reap huge profits. Although people can buy deflazacort in Canada and the European Union for about $1,200 a year, Marathon announced it would sell it under the brand name Emflaza for a new list price: $89,000.

The announcement drew a strong and swift response from U.S. Sen. Bernie Sanders, I-Vt. and Rep. Elijah Cummings, D-Md.

“We believe Marathon is abusing our nation’s ‘orphan drug’ program,” Sanders and Cummings wrote in a letter to Marathon’s Chairman and CEO Jeff Aronin. “We are investigating how Marathon set its price for this drug and how much the company stands to make as a result.”

Aronin responded in an Open Letter to the Duchenne Community posted on Marathon’s website:

“We’ve heard both support from the community and concerns about how the pricing and reimbursement details will affect individual patients and caregivers…” he wrote. “We are pausing our commercialization efforts in order to meet with Duchenne community leaders and explain our commercialization plans, review their concerns, discuss all options, and move forward with commercialization based on the resulting plan of action.”

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