Catalyst Defends $375,000 Price Tag for Previously Free Drug

Regulatory NewsRegulatory News | 21 February 2019 |  By 

In response to questions from Sen. Bernie Sanders (I-VT) on the price tag of a new orphan drug, Catalyst Pharmaceuticals on Thursday explained how much it had to invest in Firdapse (amifampridine) to bring it to market and how the previously experimental version of the drug should not be the standard of care.

Firdapse is the first drug approved by FDA to treat Lambert-Eaton myasthenic syndrome (LEMS), a rare autoimmune disorder that affects patients' muscles and nerves.

Sanders took issue with the company’s decision to set the drug’s annual list price at $375,000, saying it is “not only a blatant fleecing of American taxpayers, but is also an immoral exploitation of patients who need this medication.” He also noted that the estimated cost to manufacture the drug is $1,600-$6,000 per year.

In its defense, Catalyst said it spent “millions of dollars” to conduct two Phase 3 trials and bring the drug to market. The company also said that more LEMS patients will be able to access the drug now that it is FDA-approved and because the company has an “array of financial assistance programs” to reduce patient co-pays (“$10 or less”) and deductibles “to a nominal affordable amount.”

Catalyst also questions how using a drug not approved by FDA can be the standard of care, explaining that the use of an investigational new drug (IND) application to provide access to the drug for nearly 30 years is a cumbersome and difficult way for LEMS patients to use the treatment.

As far as why the company has decided to charge $375,000, the firm added: "We believe the pricing of our product is in line with the pricing of other products that provide significant clinical benefits in treating an ultra-orphan disease of similar severity."



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