Drug Company Walks Back Rollout of Deflazacort After Outcry

Marathon Pharmaceuticals provided an open letter to the public on Monday, February 13, 2017, explaining that the previously planned rollout of the drug deflazacort for the symptomatic treatment of Duchenne muscular dystrophy has been “paused” amid outcry from the public and some elected officials over its $89,000/year price tag.

The U.S. Food and Drug Administration approved Marathon’s deflazacort, brand name Emflaza, on February 9, 2017, the first time the corticosteroid drug that has been available for decades for the treatment of other conditions, was approved for use in the treatment of DMD in the United States.

Marathon had anticipated the commercial sale of the drug to soon follow the federal agency’s approval but has since reconsidered that decision in the wake of concerns by patients and caregivers about Emflaza’s price tag, as the company’s chairman and CEO, Jeff Aronin, explained in his open letter to the public.

Elected officials Representative Elijah Cummings (D-Maryland) and Senator Bernie Sanders (I-Vermont) sent their own letter of concern to Marathon on Monday, asking why patients in the U.S. Would face a price tag for deflazacort of $89,000/year when the same medication is available in other countries for $1,600/year or less. In their letter to Marathon, Cummings and Sanders wrote:

“We believe Marathon is abusing our nation’s ‘orphan drug’ program, which grants companies seven years of market exclusivity to encourage research into new treatments for rare diseases — not to provide companies like Marathon with lucrative market exclusivity rights for drugs that have been available for decades.”

Whether Marathon Pharmaceuticals will also face the ire of Senator Charles Grassley (R-Iowa), Chairman of the Senate Judiciary Committee, in his recently launched investigation into possible abuses of the Orphan Drug Act by pharmaceutical companies remains to be seen.

Marathon cites the cost of research and development of deflazacort for FDA approval in the United States as its rationale for the medication’s price tag, pointing out that not only is the medication now approved and available throughout the United States for the treatment of DMD in patients five-years-old and older, but much more is now known about appropriate dosing, potential side effects and drug interactions as part of the clinical trials processes.

Fewer than 10 percent of DMD patients have been importing deflazacort from other countries where the drug has been widely available. Marathon’s Aronin wrote:

“I want to assure caregivers and others that 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. We believe that access to deflazacort in the United States will be dramatically expanded once EMFLAZA is commercially available.

“I also want you to know that we hear and understand your concerns around the price and recognize it is tied to fears you have about reimbursement for other components of Duchenne treatment. We know all parents want the very best care possible for their child and being able to access quality care is paramount. We will continue the discussion on pricing and access with you until there is a full understanding of how pricing decisions directly impact the revenues needed to fund past, current and future research as well as opportunities to purchase new therapeutic treatments.”

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