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Stung by criticism of its plan to charge a whopping $89,000 a year in the U.S. for a muscular dystrophy drug that costs less than $1,500 elsewhere, Marathon Pharmaceuticals on Monday said it was "pausing" that rollout for an unspecified amount of time.
Marathon also said that American patients who now receive that drug, deflazacort, from other sources "will continue to have that option."
Those patients who suffer from the genetic disorder or their parents have been importing deflazacort from other countries, for much less than what Marathon last week said it would begin charging for it in the United States.
And Marathon CEO Jeffrey Aronin also said the company "will meet with caregivers and explain our commercialization plans, review their concerns, discuss all options, and move forward with commercialization based on an agreed plan of action."
Marathon's announcement came hours after the company was blasted by two members of Congress for its "outrageous plan" to charge $89,000 per year for Emflaza, its brand name for deflazacort, a corticosteroid that received regulatory approval for U.S. sales last week. Deflazacort used to treat Duchenne muscular dystrophy, a progressive muscular degeneration condition that afflicts fewer than 15,000 people, mostly boys.
"Since last week's approval, we have heard both support from the community, and concerns about how the pricing and reimbursement details will affect individual patients and caregivers, such as how it effects coverage of other Duchenne products, such as EXONDYS 51," Aronin wrote in a post on CureDuchenne.com, a patient advocacy website.
"Based on these questions, today we are announcing: We are pausing our launch which has not taken place yet."
"We have not sold any new product, and we will pause that process," Aronin wrote. The company didn't say when it expected to make the drug commercially available. The company didn't say when it expected to make the drug commercially available.
Marathon will also maintain an expanded access program that provides the drug for free to 800 patients, Aronin wrote.
The company followed up later Monday with an open letter to the Duchenne community, saying the resources it invested in getting the drug approved in the U.S. "were substantial and we don't expect to recoup our investment for several years."
Marathon pledged, if it's profitable, to reinvest earnings from Emflaza into more research in Duchenne muscular dystrophy, and said it believes access to the drug "will be dramatically expanded" once it's commercially available in the U.S.
Christine McSherry, a Massachusetts woman whose son Jett uses imported deflazacort, said Marathon's "pause" is "what you do in football when it's the fourth quarter and there's only 30 seconds left in the game and you want to throw off the defense."
"They're scared," said McSherry. "They're all of a sudden realizing this community is bigger than they thought it was."
She said she told Marathon's "commercial and marketing person that their drug was like giving Tylenol to a cancer patient because it is – it doesn't cure the disease."
McSherry's son takes the recently approved Exondys 51, which aims to help patients create the missing protein that leads to Duchenne. She clarified that in her analogy she was referring to a treatment that affects the progression of disease, rather than a cure.
"You can't charge this much money for a drug that's not disease modifying," McSherry said.
Marathon, in its letter, said it expects patients in the U.S. to pay "a standard co-pay of typically $20 or less per prescription," and that it has "developed the most robust patient access programs allowed by law."
Senator Bernie Sanders and Representative Elijah Cummings, who have previously eyed Valeant, Turing, and Mylan, among other pharma firms, on Monday wrote a letter to Marathon seeking answers about its drug and expressing outrage about the planned price.
"Deflazacort has been available to patients in the European Union and Canada for many years, and patients in the United States have been importing it for decades," Sanders and Cummings wrote Aronin.
"This drug is currently available in Canada and the United Kingdom for approximately $1,000 per year."
Because it's been available outside the U.S. at such lower prices, many have said that Northbrook, Illinois-based Marathon's price for deflazacort is unjustifiably high.
Sanders, an independent from Vermont, and Maryland Democrat Cummings called the price "unconscionable" in their letter Monday and asked Marathon to "significantly lower" the price before the drug hits the market.
On top of that, they asked for information by Feb. 27 on how Marathon set the price and how much it stands to make from it.
"Marathon did not develop deflazacort," the congressmen continued in their letter.
"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 to sell the drug in the United States."
As a result of the approval, Marathon also gets seven-year market exclusivity on the drug, reserved for medicines for rare diseases, as well as a priority review voucher, which can be applied to speed up FDA review of a future medicine.
Marathon can use the voucher itself or sell it under a system designed to incentivize development of medicines for rare diseases, also known as orphan drugs. The program has proven popular; in 2015, drugmaker AbbVie purchased a priority review voucher from United Therapeutics for $350 million.
Sanders and Cummings also said in their letter Marathon is "abusing our nation's 'orphan drug' program," which they said is designed "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."
Senator Chuck Grassley separately last week said he's investigating possible misuses of the orphan drug program and how they might affect drug prices.
Sanders and Cummings have investigated Marathon before, writing a letter in October 2014 to the company about its price increases for two heart medicines it had acquired, Isuprel and Nitropress. If those sound familiar, it's because, after increasing their price tags, Marathon sold the drugs to Valeant, which increased them further, according to the congressmen's letter.
"We remain gravely concerned about these recurring abuses in the pharmaceutical industry," Sanders and Cummings told Marathon in their new letter.
"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."
The latest outcry has also created an awkward situation for industry lobbying group PhRMA, which has sought over the last year and a half to publicly distance itself from actors like Martin Shkreli's Turing Pharmaceuticals and Valeant, other targets of drug pricing ire.
PhRMA CEO Stephen Ubl, on CNBC's Squawk on the Street Jan. 23, said much of the public focus on drug pricing has been on "some guy in a hoodie," ostensibly referring to 33-year-old Shkreli, and that the focus instead should be "less hoodie, more lab coats."
Shkreli responded on a website he created called Pharma Skeletons, where he said of Marathon, "These guys invented price increases. I literally learned it from them."
Marathon CEO Aronin is a member of PhRMA's board. PhRMA didn't immediately respond to a request for comment.