February 16, 2017
By Alex Keown, BioSpace.com Breaking News Staff
WASHINGTON – A harsh critic of high drug prices, U.S. Sen. Bernie Sanders has put Illinois-based Marathon Pharmaceuticals square into his sights over the pricing of their newly approved treatment for patients with Duchenne muscular dystrophy.
Earlier this month, privately-held Marathon won approval from the U.S. Food and Drug Administration for Emflaza (deflazacort) to treat patients five years of age and older, regardless of their genetic mutation. Emflaza is the first drug approved in the U.S. for those patients. However, the problem, as Sanders and other critics see, is the price that Marathon plans to charge U.S. customers for the corticosteroid—$89,000. What sparked the outcry over the price of Emflaza is the drug has been available in Europe for between $1,000 and $1,500.
Sanders, along with U.S. Rep. Elijah Cummings, another critic of high drug prices, called Marathon’s pricing “unconscionable.”
“Exorbitantly pricing potentially life-saving medications that should be widely available for a fraction of the price hinders patient access and drives up costs for the entire health care sector,” Sanders and Cummings said in a letter to Marathon.
This is not the first company Sanders has targeted for its pricing. In October, Sanders called out Ariad Pharmaceuticals for systematically increasing the price of its leukemia drug, Iclusig, more than 73 percent since the drug’s launch in 2012.
Duchene is a fatal genetic disease. Most patients are diagnosed by the time they reach five years of age and live until their mid-20s. Duchene is a rare disease, so it qualifies for orphan status under the FDA. Most drugs developed to treat orphan diseases come with a high price tag.
Sanders though, said Marathon was “abusing” the orphan drug program so it could have “market exclusivity” for a drug that has been “available for decades.”
Although available elsewhere, Marathon acquired the U.S. rights to deflazacort and conducted their own clinical trials to meet FDA approval standards—vwhich was costly. Jeff Aronin, chairman and chief executive officer of Marathon, said in an open letter to the Duchene community, that the company invested “substantial” resources into developing the drug and does not expect to recoup its investment for several years. He added that Marathon only has seven years of market exclusivity to sell Emflaza in the United States.
“When we set the price of Emflaza, we considered the following: the resources we invested to get to this point, the resources needed to complete the phase IV clinical studies and FDA post-marketing study commitments, the ability to fund future research and development, and the ability to ensure broad patient access through insurer reimbursement and our own patient assistance programs. There is confusion that this is a generic drug. In the United States, FDA considers deflazacort a new drug and we had to get it approved. Our tablets are manufactured in the United States,” Aronin said in his open letter.
CureDuchenne, a nonprofit organization focused on funding research into finding a cure for Duchenne muscular dystrophy, praised Marathon for its R&D effort concerning Emflaza.
“For the first time all those impacted by Duchenne muscular dystrophy have a FDA-approved treatment available to them,” the organization said in a statement issued after the FDA granted approval.
That criticism of its pricing for Emflaza caused Marathon to pause its commercialization efforts so the company can explain its plans, review any concerns about the pricing, discuss options and move forward. Aronin did not provide a timeline for how long commercialization efforts will be paused, but said the company remains committed to using the revenues from Emflaza into additional research into Duchenne.
