According to a publication from KSPR 33, a drug that was once given away free of charge on a provisional basis to people with Lambert-Eaton myasthenic syndrome (or LEMS) is getting a $375,000 annual price hike.

The sudden change stems from a complicated game of legal checkers between the family-run Jacobus Pharmaceutical and the publicly-traded Catalyst Pharmaceuticals.

About Lambert-Eaton Myasthenic Syndrome

Lambert-Eaton myasthenic syndrome is an autoimmune disease – a condition where the immune system mistakenly targets the body’s own tissues as threats.

In LEMS, the immune system affects the calcium channels where the nerves and muscles meet. These channels are instrumental in releasing acetylcholine into the muscles – a chemical signal that triggers contraction. This leads to critically low acetylcholine levels in affected individuals, leaving their muscles unable to effectively contract. Less acetylcholine, weaker muscles.

It most typically affects the upper legs, upper arms, and hips. It can eventually lead to problems with walking. In some cases facial muscles might be affected, but these symptoms are typically mild.

Jacobus Pharmaceutical

Jacobus Pharmaceutical is a mom-and-pop pharmaceutical company, if ever there was such a thing.

Several years ago, Jacobus discovered that a drug called 3,4-DAP (short form for 3,4-diaminopyridine) showed promising results in treating LEMS symptoms. However, given that it can cost hundreds of millions of dollars and decades of paperwork to get a drug approved by the Food and Drug Administration, Jacobus initially declined to apply.

Despite that, some people with LEMS were still able to get their hands on the drug. If you do enough paperwork, see enough doctors, and take enough tests, certain allowances can be made to permit access to drugs not approved by the FDA.

David Tablish was one of those people, and he calls the 3,4-DAP a “miracle drug”.

“We were just elated that he could be halfway normal,” his wife Laveta remembered.

Taking into account the rarity and severity of LEMS, Jacobus Pharmaceutical for years had a policy of sending the drug to patients across the country free-of-charge.

Catalyst Pharmaceuticals

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After several years of giving 3,4-DAP away with no charge, Jacobus finally began to move on getting their drug approved by the FDA. Their hopes were dashed, though, when competing Catalyst Pharmaceuticals applied for and received approval for a modified form of the drug – Firdapse.

Because of how rare LEMS is, Firdapse also received an orphan drug designation. This designation is sometimes applied by the FDA to drugs that will only treat a very small number of Americans each year (under 200,000), and it comes with a lot of features.

The company responsible for the “orphan” drug is entitled to numerous benefits including tax breaks and reimbursements. Most significantly, though, they are granted a seven-year window of market exclusivity – in which no competitor may market a competing version of the same drug.

That means that when Firdapse won its FDA approval, Jacobus Pharmaceutical lost its baby – the company is no longer permitted to distribute 3,4-DAP.

Catalyst Pharmaceuticals’ approach isn’t nearly as pro-bono as Jacobus’ – the price tag for Firdapse is a walloping $375,000 a year. The company emphasizes that this figure isn’t representative of what patients’ co-pays might look like. In reality, patients are likely to only pay a fraction of this price – the difference, however, will be made up by the insurance companies.

While Catalyst claims that their FDA approval is good for 3,4-DAP and people living with LEMS, David and Laveta Tablish aren’t so sure. They worry that the VA providing David’s healthcare might not agree to cover the cost of the drug.

“It’s just stressful because we have to go through all this again. We thought we were settled in… all of a sudden it’s like we’re going back in time trying to explain ourselves again and why we need this help,” Mrs. Tablish said.

Do you think Catalyst Pharmaceuticals is right, and that FDA approval for their form of 3,4-DAP was good for LEMS patients? Or do you think it would have been better if Jacobus continued to give the drug away for free, but unapproved? Share your side with Patient Worthy!