Catalyst Pharmaceuticals CEO Patrick McEnany defended the price of its LEMS drug Firdapse in response to a letter sent to the company by Senator Bernie Sanders asking for an explanation as to why it was set so high. Firdapse is approved to treat patients with Lambert-Eaton myasthenic syndrome (LEMS), a rare neuromuscular disorder that makes it difficult to move the muscles normally.

The active ingredient in Firdapse, amifampridine phosphate, had been available for free for many years under a compassionate use program before Catalyst won U.S. Food and Drug Administration approval to market a modified version of the drug late last year, and announced a list price of $375,000 when it was launched in January 2019. Sanders called the price egregious and promised to call on Congress to act if it did not reduce the price substantially.

Catalyst’s response included a summary of why it will not reduce the price and wrote that it had developed several financial assistance programs, so “in most cases the patient’s monthly out-of-pocket expense will be $10 or less.”

Catalyst maintains that it will be fulfilling a large unmet need. Previous to the FDA approval only about 200 patients were receiving the experimental drug out of about 3000 LEMS patients in the United States. Now, the company wrote, all patients would be able to take an FDA-approved therapy, and be assured of its safety and efficacy.

The company justified the cost of Firdapse writing that it conducted 70 studies, clinical and non-clinical, including two late-stage studies, which cost the company millions of dollars, though it did not break out the exact amount. The FDA has also required it to conduct post-market studies and the company is evaluating Firdapse as a treatment for other rare neuromuscular diseases.

Catalyst maintained that its pricing is consistent with the pricing of other ultra-orphan disease therapies and expects the net sales price will be 15 percent to 20 percent lower that the list price.

McEnany closed his letter by encouraging Sanders to refer LEMS patients and physicians he has spoken with to contact him personally. “I will do everything I can to help make their journey as stress free as possible,” he wrote. “That is what we are committed to doing and have been doing at every turn.”

It’s unclear how Sanders will respond, but given his recently announced 2020 presidential bid, drug pricing and the Catalyst case will likely remain in the spotlight.

Photo: Patrick McEnany, CEO of Catalyst Pharmaceuticals