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Catalyst Announces Expanded Access For Myasthenic Syndrome Drug

Shaw, Gina

doi: 10.1097/01.NT.0000451007.63836.f7
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Catalyst, a Florida-based pharmaceutical company, has agreed to make available its version of a drug for Lambert-Eaton myasthenic syndrome to patients for free under a compassionate care program. Some experts are concerned, though, that the price for the drug could spike if and when it is approved for use by the US Food and Drug Administration.

Under fire for its pursuit of Food and Drug Administration (FDA) approval for its version of a drug for Lambert-Eaton myasthenic syndrome (LEMS) — a drug that has been provided at no cost for two decades by another pharmaceutical company under the FDA's compassionate use program — Catalyst Pharmaceutical Partners announced in April that it will offer the investigational potassium channel inhibitor, known as amifampridine (Firdapse), to LEMS patients at no cost “until sometime after approval.”

“As part of our ongoing commitment to the LEMS community, we are making [amifampridine] available to those who would like access to this investigational drug, but are unable to participate in clinical studies or travel to sites where it is available,” said Catalyst Chief Executive Officer Patrick J. McEnany.

Late last year, Neurology Today reported on the controversy surrounding amifampridine. [See “Free Myasthenic Syndrome Drug Program Threatened in Race for FDA Approval,”]

For more than 20 years, the New Jersey-based, family-owned Jacobus Pharmaceuticals has been supplying some 600 LEMS patients with 3,4-diaminopyridine (3,4-DAP) — the only drug known to be effective against the disorder, which causes severe muscle weakness — at no charge.

Catalyst, which is based in Florida, has signed an agreement with BioMarin Pharmaceuticals, which holds US licensing rights for the drug and which began marketing it in Europe in 2010, to underwrite clinical trials in the US — trials that are now in Phase 3.

If Catalyst beats Jacobus to FDA approval — the smaller firm has only completed Phase 2 trials — experts fear that the once-free drug will undergo price spikes similar to those seen in Europe. There, 3,4-DAP was once available from compounding pharmacies for under $2,000 annually; it now costs $60,000 a year.

McEnany said that Catalyst has been discussing a compassionate-use program of its own for amifampridine for months. “There has been anecdotal information through social media that patients who were receiving their drug elsewhere were having trouble accessing it,” he said. “There have been supply issues with getting the active ingredient from the source, which is outside the US. We were dealing with our own supply issues as well, but now we're in great shape.”



For its part, Jacobus reported that it has had no shortages and all of its compassionate-use patients have received their medication on schedule.

McEnany told Neurology Today that Catalyst's expanded access program should last between seven quarters and two years, “until we launch the drug.” He said he can't predict how many patients the company expects to serve. “We don't know the extent of the current shortage, but we do believe that there's greater demand. It could be 50 patients; it could be 500 patients.”

Ultimately, of course, Catalyst hopes to “convert” these compassionate-use patients to a commercial product after gaining FDA approval for amifampridine. Company spokesman David Connolly confirmed that as of the end of April, Catalyst had completed enrollment of the 36 patients needed for its Phase 3 trial.

McEnany said that the company expects to start a rolling submission of its New Drug Application in the first quarter of 2015, and complete the submission in the second quarter of the year, with an eye toward an early 2016 launch.

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What will that mean for patients? Yadollah Harati, MD, FAAN, a professor of neurology and director of the Neuromuscular Section at Baylor College of Medicine and past chair of the Neuromuscular Section of AAN, is worried.

“This reminds me of what happened with another drug, pyridostigmine, for myasthenia gravis,” said Dr. Harati, who is one of the lead investigators for the Jacobus trial, which has enrolled 30 patients.

“It was very cheap and widely available. All of a sudden, one of the companies got hold of the marketing and the price suddenly went up 250 percent.” (That pricing pressure eased after the drug, Mestinon, went generic in 2005.) “It makes me concerned that we may be heading in that direction, with a drug that has been so cheap and a company that so generously has been providing it to all patients free of charge, suddenly becoming very expensive as it has been in Europe.”

In general, Dr. Harati said, competition is better for patients. “The ALS drug riluzole [Rilutek] has no competition, and it's still very expensive even after so many years in the market.” (Without insurance or other third-party coverage, the cost can be $1200 a month.)

“I like to see more competition, provided that the drugs are equally effective, but on the other hand, I am afraid that if one company gets hold of the approval and jacks up the price, patients may suffer.”

McEnany said that Catalyst plans to offer a patient assistance program after the drug is approved. “As with most orphan drugs, we would set up a program to provide copay help for those in financial need. We've conducted some market access research and concluded that third-party payers, both public and private, understand the need. We want to make sure we fill in the insurance gaps as well as possible.”

“This is a free country and people can compete,” Dr. Harati concedes. “But if the two drugs are identical in terms of efficacy, I don't see any reason for a second drug, especially since this compound has so far been provided at no charge. I can't speak for Jacobus, but I have the feeling that if they get FDA approval first, they are not going to increase the price.”

Neurology Today reached out to Jacobus representatives, but they said they had no comment on Catalyst's decision to offer an expanded-access program.

Dr. Harati noted that one of the eight patients in his arm of the trial flew from Argentina to participate, because the price of the drug there had skyrocketed as it has in Europe. “Our US participants in the trial knew about the price increases in other countries and were very concerned that this may happen here as well.”

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•. The Catalyst compassionate care program for amifampridine: Call toll-free 1-844-FIRDAPSE (1-844-347-3277)
    •. The FDA expanded access program:
      •. Firdapse clinical trial enrollment:
        © 2014 American Academy of Neurology