A Northbrook pharmaceutical company is "pausing" its commercial launch of a newly approved drug for muscular dystrophy -- a medication with an $89,000 price tag that has shocked patient advocates and members of Congress.

Marathon Pharmaceuticals' deflazacort, used to treat patients with a fatal muscle wasting disease called Duchenne muscular dystrophy, gained approval from the U.S. Food and Drug Administration last week.

But many have criticized its $89,000-a-year price, noting the drug has been available outside the U.S. for years, and some Americans have been importing it for as little as $1,200 a year.

Marathon CEO Jeffrey Aronin said in a letter Monday to the Duchenne community, posted on the company's website, that he expected patients would pay a standard copay of $20 or less per prescription — and Marathon had good reasons for pricing it at $89,000.

But he also said Monday the company would pause its "commercialization efforts" in order to meet with "community leaders" to further discuss the issue.

The about-face followed a scathing letter sent to Marathon on Monday by Sen. Bernie Sanders, I-Vt., and Rep. Elijah Cummings, D-Md., in which they called the pricing "outrageous."

"Exorbitantly pricing potentially life-saving medications that should be widely available for a fraction of the price hinders patient access and drives up cost for the entire health care sector," they wrote.

FDA approves Northbrook company's $89,000 muscular dystrophy drug Lisa Schencker

The U.S. Food and Drug Administration on Thursday approved a Northbrook pharmaceutical company's drug to treat a deadly form of childhood-onset muscular dystrophy — only the second FDA-approved drug for the disease and the first for everyone with the disorder.

Marathon Pharmaceutical's deflazacort,...

The U.S. Food and Drug Administration on Thursday approved a Northbrook pharmaceutical company's drug to treat a deadly form of childhood-onset muscular dystrophy — only the second FDA-approved drug for the disease and the first for everyone with the disorder.

Marathon Pharmaceutical's deflazacort,...

(Lisa Schencker)

"Marathon's apparent abuse of government-granted exclusivity periods and incentives to sell what should be a widely available drug for $89,000 a year is unconscionable."

Babar Ghias, Marathon chief financial officer, last week told the Tribune the price was low compared with other drugs treating rare diseases that affect a similar number of patients. The top 100 orphan drugs — medications used to treat rare diseases — in the U.S. cost an average of $111,820 a year per patient in 2014, according to a report by Evaluate, a market research firm.

In his letter Monday, Aronin said Marathon set the price at $89,000 based on the resources it invested to bring the drug to market and complete clinical studies, as well as to fund future research and ensure broad patient access through insurer reimbursement and its own assistance programs.

But Sanders and Cummings accused Marathon of "abusing" the nation's orphan drug program, in which companies get exclusive rights to sell a drug for seven years. The idea behind such exclusivity is to encourage research into treatments for rare diseases, not to reward companies for bringing drugs to market that already have been available for decades, the lawmakers wrote.

Liam McNicholas Alyssa Pointer / Chicago Tribune Liam McNicholas, 12, of Lindenhurst, talks with Meghan Kostyk, an advanced practice nurse, on Feb. 9, 2017, during his biannual visit to Lurie Children's Hospital for a checkup. Liam has Duchenne muscular dystrophy, a condition which is treated by a drug from a Northbrook company that just received FDA approval. Liam McNicholas, 12, of Lindenhurst, talks with Meghan Kostyk, an advanced practice nurse, on Feb. 9, 2017, during his biannual visit to Lurie Children's Hospital for a checkup. Liam has Duchenne muscular dystrophy, a condition which is treated by a drug from a Northbrook company that just received FDA approval. (Alyssa Pointer / Chicago Tribune)

They said they're investigating how Marathon set the drug's price and how much it stands to profit from it.

Aronin defended that exclusivity period in his letter, writing, "The resources we invested were substantial and we don't expect to recoup our investment for several years and we have only 7 years of market exclusivity. ...

"We hear and understand your concerns around the price," Aronin wrote.

Christine McSherry, founder of the Massachusetts-based Jett Foundation, said she was pleased Monday to hear the company would take a step back.

"I think everybody was shocked by the price," said McSherry, whose 21-year-old son, Jett, has Duchenne. "We have questions about how they came to that amount and how they justify it."

The uproar over deflazacort is the latest outcry over drug prices — an issue that ignited concern at the highest levels of government.

Duchenne muscular dystrophy is a genetic disorder that affects about 15,000 patients, mostly boys, in the U.S. The disease can cause movement, heart and breathing problems and those with it typically lose the ability to walk, and die in their 20s or 30s. Deflazacort, which will be sold in the U.S. under the brand name Emflaza, is a steroid meant to improve muscle function, so those with the disease might be able to feed themselves, control their wheelchairs or walk longer.

It's the second drug approved by the FDA for treating Duchenne — the first one was just approved last year. Many patients also have been using a different steroid called prednisone, though prednisone is not specifically approved for treating Duchenne.

lschencker@chicagotribune.com

Twitter @lschencker

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