Last month, an article in TheStreet.com levied some harsh allegations against Catalyst Pharmaceuticals Partners and its pursuit of FDA approval for the orphan drug, Firdapse.
Firdapse treats Lambert-Eaton Myasthenic Syndrome (LEMS) a rare, progressive disorder that is characterized by muscle weakness, and often associated with lung cancer. Currently, Firdapse is being evaluated in a Phase 3 clinical trial and, if the trial results are successful, Catalyst expects to submit a new drug application (NDA) with the FDA in the first half of 2015.
The issue, however, as pointed out by TheStreet, is that for the past 20 years, Jacobus Pharmaceuticals, a small, private, family-owned pharmaceutical company in New Jersey, has provided LEMS patients in the U.S. with an effective drug known as 3,4-Dap free of charge. And, according to Jacobus, 3,4-Dap and Firdapse are equivalent. (Analysts guesstimate Firdapse pricing to exceed $60,000 per year.)
"Firdapse is not a new compound. It's the same drug we make. What Catalyst is doing is not the same as a company profiting from a new invention. What Catalyst is doing is making money off LEMS patients. They don't want to help LEMS patients, they just want to make money. If I worked for Catalyst, I wouldn't be able to sleep at night," says Laura Jacobus.
Catalyst CEO, Patrick McEnany, disagrees: "Firdapse contains the phosphate salt of 3,4-Dap. This pharmaceutical drug substance is more stable than the free base form of the 3,4-Dap that is available from Jacobus. Catalyst believes that Firdapse will have superior stability at room temperature which will provide patients with the assurance that their drug has the correct potency and purity throughout its shelf life, even when stored in a medicine cabinet or carried on the patients person for an extended period of time. Catalyst's Firdapse tablets, and its active ingredient, are produced on a commercial scale using validated processes and under all requirements of GMPs (this is a requirement for phase III clinical trial medications."
Catalyst also addresses the issue in it's October 29 update by saying "Catalyst has previously reported that another pharmaceutical company is conducting a Phase 2 trial with a different formulation of amifampridine (3,4-DAP) for the treatment of LEMS. While there can be no assurance, Catalyst continues to believe that its development program for the product is further along in the development than this other company."
So, the race to complete the study and submit the data to the FDA for approval is on. What do you think?