- The Washington Times - Monday, February 13, 2017

An Illinois company says it will pause the rollout of its muscular dystrophy drug after two members of Congress demanded to know Monday why it planned to charge $89,000 per year, even though it’s been widely available overseas for just $1,000.

Sen. Bernard Sanders, Vermont independent, and Rep. Elijah E. Cummings, Maryland Democrat, said the price is “unconscionable,” making Marathon Pharmaceuticals the latest drugmaker to attract the ire of Capitol Hill lawmakers who think too many companies are gouging U.S. customers for profit.

The Food and Drug Administration last week approved defalzacort, a drug used to treat the severe Duchenne form of muscle dystrophy that affects 15,000 people in the U.S. — for use in the states under the brand name Emflaza.

Mr. Sanders and Mr. Cummings said they are upset that Marathon, which didn’t develop the drug, plans to charge Americans far more than people in the U.K. or Canada, arguing the company acquired old clinical trial data and performed “some additional analyses” to win approval from the FDA.

“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,” wrote Mr. Cummings, the top Democrat on the House Oversight Committee, and Mr. Sanders, a self-described socialist who attracted a massive following during his 2016 Democratic primary campaign.



In an open letter to Duchenne patients, Marathon CEO Jeff Aronin said the company’s initial meetings with insurers and other payers had gone well, and that many felt the price was “appropriate given the very small patient population.”

Mr. Aronin said the company poured substantial resources into the approval process, and that it doesn’t expect to recoup that investment for several years. It has seven years of market exclusivity.

Mr. Aronin also said only 7 percent to 9 percent of the patient population had imported the drug for use prior to FDA approval. He said patients can continue to access the drug that way while the company pauses to explain their marketing plans to the patient community and listen to their feedback.

“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,” Mr. Aronin wrote. “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.”

Yet skeptical lawmakers hadn’t been satisfied with another Marathon executive’s earlier defense to The Wall Street Journal — that deflazacort was “modestly priced for an orphan drug.”

“That argument is not a defense of Marathon’s actions, but rather an indictment of a system that allows drug companies to engage in such opportunistic pricing behavior,” they wrote.

The lawmakers want to know how much it cost Marathon to acquire the drug and get it approved, and how much they expected to get in terms of revenue and profit.

Their scrutiny comes several months after Congress lit into Mylan — the maker of EpiPens that deliver life-saving relief to those who go into allergic shock — for raising the price of its twin-packs from $100 in 2009 to more than $600 last year.

The wave of scrutiny, including a Senate probe and House hearings, paved the way last year for massive civil settlement and a half-price generic version of the epinephrine injectors.

High-profile hearings also turned then-CEO Martin Shkreli into a national punching bag for hiking the price of a drug from $13.50 to $750 during his tenure at Turing Pharmaceuticals, while the chief of Valeant Pharmaceuticals International publicly admitted to “mistakes” for buying the rights to drugs in order to hike their prices and turn a profit.

Mr. Sanders and Mr. Cummings are working on legislation that would let Medicare, the government insurance program for seniors, negotiate with drugmakers for lower prices.

They’ve urged President Trump, who has sent mixed signals on pharmaceutical pricing, to partner with them on the effort.

Mr. Trump recently said the U.S. “does not bid properly for drugs,” giving Democrats hope he’d use government authority to bring down prices, though he’s also signaled support for GOP-favored reforms, such as speeding up approval times for new medicines.

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