Controversial List Price Puts Muscular Dystrophy Drug Launch on Hold

Just days after the US Food and Drug Administration (FDA) approved Marathon Pharmaceuticals’ Duchenne muscular dystrophy (DMD) drug, Emflaza, the company has already faced criticism over the drug’s $89,000 per-year price tag. Emflaza is the first steroid drug to be approved to treat the rare disorder, and only the second ever drug to be made available to this patient group.

Though rebates and financial assistance promised by Marathon could bring the cost of the medication down to $54,000 per year, this is much more expensive than some patients were originally paying. Before now, this drug was unapproved by the FDA however a select group of patients imported the generic version – deflazacort – from Canada and the UK for approximately $1,000 for one year of treatment.

“As we all know, deflazacort was being used without FDA approval — and without ever having been approved anywhere in the world for Duchenne — by a very small group of patients (roughly 7 to 9 percent) in the Duchenne community who imported it from overseas,” wrote Marathon’s chairman and CEO, Jeff Aronin, in an open letter to the Duchenne community. “Our goal in commercializing Emflaza all along has been to make it available to a much broader set of patients who, prior to FDA approval, have not had access to this therapy. There was also much we did not know about using the drug in the Duchenne patient population including proper dosing, potential side effects and drug-to-drug interactions.”

But despite Marathon’s investment into 17 preclinical and clinical studies investigating deflazacort use in patients with DMD, the company still faced criticism over their published list price. “Marathon did not develop deflazacort,” wrote Senator Bernie Sanders and Representative Elijah Cummings in a letter to Aronin. “Rather, Marathon acquired the rights to historical clinical trial data from the 1990s and completed some additional analyses to gain approval from the FDA to sell the drug in the U.S.”

Along with asking Aronin to provide documents to support their drug pricing decision, Sanders and Cummings also accused Marathon of abusing the FDA’s orphan drugs program. According to the Democrats, the program is meant to encourage research into new therapies for rare diseases by granting a seven year period of market exclusivity to the pharmaceutical company. They say it’s “not to provide companies like Marathon with lucrative market exclusivity rights for drugs that have been available for decades.”

For now, Aronin has suspended commercialization efforts for Emflaza, while maintaining their expanded access program for the drug. Aronin also promised that patients obtaining deflazacort through alternative channels – such as importation – will continue to be able to do so, however it’s unlikely that this option will be available for long.