Nearly three weeks after receiving a letter from Senator Bernie Sanders, Catalyst Pharmaceuticals (CPRX) defended its pricing for Firdapse, a medication for the treatment of adults with Lambert-Eaton myasthenic syndrome.
BACKGROUND: On February 4, Senator Bernie Sanders sent a letter to Patrick J. McEnany, chairman and chief executive officer of Catalyst Pharmaceuticals, regarding the $375,000 price tag for Firdapse, a recently-approved drug for Lambert-Eaton myasthenic syndrome, or LEMS. In November 2018, after a previous rejection, Catalyst won approval from the U.S. Food and Drug Administration for the oral drug as a first-ever approved treatment for LEMS, which affects the connection between nerves and muscles and causes weakness and other symptoms in affected patients. It affects approximately one in 100,000 people in the United States and about 3 million people globally, Catalyst said in November.
Sanders wrote in his letter that for two decades, LEMS patients were able to receive the same drug, known as 3,4-DAP, for free from Jacobus Pharmaceutical under the Food and Drug Administration’s compassionate use program. Recently, Catalyst licensed the rights to the drug and received exclusive rights to market Firdapse for seven years under the FDA’s orphan drug designation. In December 2018, Catalyst announced to investors it would set the list price for Firdapse at $375,000 per year. As a result, Sanders said patients around the country "are frightened as to whether or not they will be able to maintain access to a drug they depend upon to survive." In response to the concerns raised by patients, Sanders asked the CEO of Catalyst how many patients will suffer or die due to their decision to set such an outrageous price, calling that decision "not only a blatant fleecing of American taxpayers, but...also an immoral exploitation of patients who need this medication." "By setting such a high price and forcing production and distribution of the older, inexpensive version to cease, you are threatening access that patients had to a cheap version of this product, and handing a completely unwarranted bill to American taxpayers," Sanders wrote.
Sanders requested information on what Catalyst is charging patients, private insurers and government payers for the medication. "The egregious price set by Catalyst cannot be allowed to stand. Patients in America should not be allowed to suffer or die because of the greed of a drug company. If Catalyst does not substantially lower the price of this medication, Congress must act to ensure it is affordable for every patient," Sanders said.
CATALYST DEFENDS FIRDAPSE PRICING: In response to Sanders, Catalyst said it "respectfully question(s)" the notion that an experimental product that hasn't received FDA approval is an acceptable standard of care for the Lambert-Eaton myasthenic syndrome patients treated by Firdapse. Further, Catalyst believes its pricing of Firdapse is "in line with the pricing of other products that provide significant clinical benefits in treating an ultra-orphan disease of similar severity." In its response, Catalyst cited the "large unmet medical need" for an FDA-approved therapy to treat LEMS patients, and said with this FDA approval, all adult LEMS patients -- and not just a few -- have "affordable" access to the drug. Additionally, Catalyst noted that it has developed "an array" of financial assistance programs that are available to help reduce patient co-pays and deductibles to a "nominal affordable amount," and added that "a physician can now simply write a prescription and Firdapse will be delivered to the patient's door within 2-3 days and in most cases the patient's monthly out-of-pocket expense will be $10 or less." It also contended that forecasted rebates, discounts, patient commercial co-pay support, Medicare coverage gap subsidies, statutory Medicaid discounts and other governmental discounts will results in the net sale price of Firdapse being 15%-20% lower than its annual list price for the product.
Catalyst CEO McEnany concluded the response to Sanders by saying that "We are carefully listening to LEMS patients and the neurologist and neuromuscular physical communities." He added that "if patients or their physicians with whom you speak are concerned, I welcome them to contact me personally and I will do everything I can to help make their journey as stress free as possible -- that is what we are committed to doing and have been doing at every turn."
PRICE ACTION: In morning trading, shares of Catalyst Pharmaceuticals are up 1.5% to $2.80.
Catalyst Pharmaceuticals
+0.04 (+1.45%)