For the past two decades, patients with the rare neuromuscular autoimmune disease Lambert-Eaton Myasthenic Syndrome (LEMS) have received a drug known as 3,4-DAP for free under the U.S. Food and Drug Administration’s compassionate use program. Now they may be faced with paying as much as $375,000 a year for essentially the same drug. And Senator Bernie Sanders (D-Vermont) wants to know why.

The active compound was approved for marketing by the FDA as Firdapse last November. In December, Catalyst Pharmaceuticals announced that it would set the list price of Firdapse at $375,000. Patients will no longer be able to get 3,4-DAP for free for at least the next seven years while Catalyst enjoys exclusivity.

As the U.S. Congress continues to look into the issue of drug pricing, Sanders has called out Catalyst over what he terms its “egregious price” for the drug. In a letter to Catalyst CEO Patrick J. McEnany, Sanders asked the company to explain step by step why it set such a high price, calling that decision “not only a blatant fleecing of American taxpayers, but…also an immoral exploitation of patients who need this medication.” 

Catalyst Pharmaceuticals licensed the North American rights to Firdapse from BioMarin Pharmaceuticals in 2012 when it was in late-stage U.S. studies and had Orphan Drug designation as a treatment for LEMS. It had already been approved for use in Europe.

In LEMS, the immune system attacks the areas where the nerves and muscles connect, affecting the way they communicate and making it difficult to move the muscles normally. Sanders considers the high price as a barrier to access and asked Catalyst to report how they plan to make it available to patients.

In the letter he pointed out that patients have reported that Catalyst will not directly offer payment assistance, instead directing them to charities. Patients have also said that the company has only approved one specialty pharmacy to distribute the drug. He wants to know if these reports are true.

“Patients in America should not be allowed to suffer or die because of the greed of a drug company,” Sanders said. “If Catalyst does not substantially lower the price of this medication, Congress must act to ensure it is affordable for every patient.”

Sanders asked for a response by February 18th. In an email statement sent to RARE Daily, Catalyst responded that its top priority is improving patient care in the LEMS community and potentially elsewhere within the neuromuscular community.

“We will respond to Senator Sanders’ letter in a timely manner and provide information about Firdapse and the programs that we have in place to raise awareness of LEMS, facilitate accurate and timely diagnosis, and broaden affordable patient access to an FDA-approved treatment,” the company wrote.

Photo: Senator Bernie Sanders

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