“Marathon did not develop deflazacort,” Sanders and Cummings wrote. “Rather, Marathon acquired the rights to historical clinical trial data from the 1990s and completed some additional analyses to gain approval from the Food and Drug Administration (FDA) to sell the drug in the U.S.”
Marathon said in the statement that it appreciates the opportunity to engage with Sanders and Cummings on the value of the drug. The company expects patients will pay a standard co-pay of $20 per prescription, Aronin said in the statement
“Our preliminary meetings with the payer/insurer community have gone well and many have acknowledged the price was appropriate given the very small patient population,” he said.
The lawmakers also accused Marathon of taking advantage of an FDA program, known as the orphan drug program, that will give it seven years of exclusive sales rights for the treatment. The lawmakers said the program was not meant to “provide companies like Marathon with lucrative market exclusivity rights for drugs that have been available for decades.”
The orphan drug program is drawing bipartisan scrutiny from lawmakers. Republican Senator Chuck Grassley announced Friday that he is exploring possible misuses of the orphan drug program and its effect on drug prices. The timing of Grassley’s inquiry and Marathon’s drug approval were coincidental, said Grassley spokeswoman Jill Gerber.
Cummings said last week that he’s scheduled to meet with President Donald Trump this week to discuss prescription drug prices. Trump has pledged to lower drug costs in the U.S., in addition to calling for allowing Medicare to negotiate with drug companies for lower prices.
Second Drug Approved
Duchenne muscular dystrophy, or DMD, is a rare genetic disease that mainly affects young boys. Patients lack a protein called dystrophin that’s essential for healthy muscle, and eventually lose the ability to walk, stand and breathe. DMD affects about one of every 3,600 male infants globally, according to the FDA.
Emflaza is an anti-inflammatory corticosteroid used to treat the symptoms of DMD, and is the second drug to be approved in the U.S. for the disease. The other is Sarepta Therapeutics Inc.’s Exondys 51, which is designed to help patients with a certain genetic profile make dystrophin. Patients also raised questions about whether using Emflaza would affect their access to Exondys 51, according to the statement.People who currently get deflazacort from other sources will continue to be able do to so during the pause, Aronin said in the statement. The company will maintain its expanded access program, he said.