Catalyst Pharmaceuticals defends $375,000 drug price after Bernie Sanders rebuke

(Reuters) – Catalyst Pharmaceuticals Inc, rebuked by U.S. Senator Bernie Sanders for its high drug prices, on Thursday defended its $375,000 treatment for a rare disease, saying the price was in line with similar products in the industry.

Sanders, a vocal critic of high drug prices, had written to Florida-based Catalyst earlier this month, asking it to justify its price for Firdapse, a medication for a rare neuromuscular disease that affects about one in 100,000 people in the United States.

Drug pricing has been a major talking point in the United States for the past few years. It was also one of the campaign promises made by President Donald Trump in the 2016 elections, but so far the debate has not resulted in lowering healthcare costs for Americans.

The Trump administration last month proposed a rule to overhaul the industry’s system of rebates, or discounts, while Sanders last month unveiled legislation aimed at lowering drug prices.

For years, patients were able to get Firdapse for free from Jacobus Pharmaceuticals, a small New Jersey-based drug company that offered the drug through a U.S. Food and Drug Administration (FDA) program called “compassionate use.”

The program allows patients with rare diseases and conditions access to drugs not yet approved outside of a clinical trial, when there is no viable alternative.

Catalyst bought the U.S. rights to the drug in 2012 and got approval to sell it in the United States in November last year.

The company said, before its November approval, only about 200 of an estimated 3,000 Lambert-Eaton Myasthenic Syndrome (LEMS) patients in the United States received some form of Firdapse, pointing to a large unmet medical need.

“Now, for the first time, LEMS patients have confidence their therapy is FDA approved and is safe and effective,” Catalyst’s Chief Executive Officer Patrick McEnany said in a letter.

The office of Bernie Sanders, who is seeking the Democratic Party’s presidential nomination in 2020, was not immediately available for comment.

The company said that Firdapse’s price was similar to other products that provide a significant clinical benefit in treating ultra-rare diseases, adding it believed the drug would be widely reimbursed by insurers for the small population it treats.

Physicians can write a prescription where Firdapse will be delivered to a patient’s door and in most cases at an out-of-pocket cost of less than $10 per month, Catalyst said.