Business Wire, AP

Utah LEMS patient shocked after cost of treatment went from being free to costing $375K a year

By Cara MacDonald, | Posted - Feb 16th, 2019 @ 9:38am

SALT LAKE CITY — A Utahn with a rare neurological disease is balking at the sight of his medical bills after FDA approval caused his free treatment to go up to $375,000 a year.

LEMS, also known as Lambert-Eaton myasthenic syndrome, is an autoimmune disease which causes the body’s immune system to attack the connections between nerves and muscles. As a result, the brain can’t communicate well with muscles, making it very difficult to move and even swallow or breathe in some cases.

Firdapse is a drug designed to treat LEMS, which affects 1 out of every 100,000 people, according to Fortune. The drug used to be free, but in December the annual cost of using it was raised to $375,000.

Connor Arrington, 31, is one LEMS patient feeling the effects from the price bump. “My relationship with LEMS started almost four years ago,” he told “It felt like I was wearing 10-pound ankle weights on both legs, and I was having a harder time moving around and just doing normal activities.”

Arrington had some initial tests done, which didn’t come up with anything. He didn’t have any vitamin deficiencies and seemed to be healthy. He assumed maybe he was just out of shape, but as years passed and his condition didn’t improve, Arrington began to do some more digging.

“A family member came across some of the symptoms for LEMS,” Arrington explained. “Whether or not it might be something I had, I figured I should probably see a neurologist.”

In the fall of 2018, he was able to see a neurologist and obtained a successful diagnosis with LEMS. “I was almost immediately started on a medication called 3,4-DAP.”

For about 20 years prior to Catalyst’s release of Firdapse, a small drug company called ‘Jacobus Pharmaceuticals’ was offering a non-Federal Drug Administration approved experimental drug called 3,4-DAP, according to Bio World. They never sought FDA approval and instead offered the drug at very little cost to patients suffering from LEMS through a compassionate use program.

The first day they had Arrington try the medication, it was only about 20 minutes before he felt a life-changing experience. “It was as if my legs grew wings," he said. "They felt as light as air. I could walk around or even run around. It was a surreal experience. I hadn’t felt that way in more than three years.”

Unfortunately, Arrington's experiences with 3,4-DAP didn’t last long. Catalyst Pharmaceuticals had been conducting clinical trials for a couple of years seeking to get an almost identical drug, Firdapse, approved by the Federal Drug Administration. They were given the green light at the end of 2018 and quickly pushed the drug to market in early 2019.

When a treatment gains FDA approval, other non-approved options are not able to be sold anymore, meaning 3,4-DAP is no longer available to LEMS patients. The new drug costs around $375,000 annually without insurance and co-pays will depend on the patient’s insurance plan, according to Fortune.

“I haven’t received final word from my insurance company (about) what kind of financial burden that will place on me, but I’ve heard other people getting quoted a $2,600/month co-pay,” Arrington said.

Sen. Bernie Sanders, D-Vermont, would like to know why. He wrote a letter to Catalyst on Feb. 4 asking them to lay out the factors leading them to set the list price so high and see how many patients might die or suffer significantly as a result, according to CNBC.

Catalyst CEO Patrick McEnany told NPR that Catalyst, together with BioMarin, has invested about $40 million in research and development of the drug. If the medicine is too expensive for a patient, he added, Catalyst will provide it at a discount or free of charge.

Information has not yet been distributed for how those discounts will be allocated, or who qualifies.

Dr. Gordon Smith, head of the division of neuromuscular medicine at the University of Utah School of Medicine, told NPR he isn't positive. "This is a drug that's cheap to manufacture, that's been available for decades," he said.

Arrington is worried he will not be able to afford the drug. As a law student at the University of Utah, he has no income and is not sure he’ll be able to pay co-pays. “It appears to me to be wholly immoral and taking advantage of individual citizens most likely not in any position to question or pay for that or try and find an alternative option,” Arrington added. “It’s paralyzing.”

Catalyst, on the other hand, believes Firdapse being FDA approved is very beneficial to patients, as it ensures what they are using has been adequately tested to governmental standards.

Dr. Perry B. Shieh, with the Department of Neurology at UCLA Medical Center, said in the Catalyst press release, “It’s important that patients living with this debilitating disease now have access to an FDA-approved treatment that has shown clinically meaningful benefits.”

Both Arrington and Sanders worry that the increased cost of the drug will not only hurt LEMS patients, but it will also distribute substantial costs among other insurance beneficiaries and raise the cost of taxes.

Firdapse was one of many drugs to experience significant price elevations in 2019, according to Fortune. As of Jan. 3, three dozen drugmakers have raised costs for more than 250 prescription drugs with an average cost increase of around 6.3 percent. For individuals being treated for LEMS, the increase is quite a bit more substantial.

Cara MacDonald

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