I wanted to update readers on Catalyst Pharmaceuticals, whom I first wrote about here. They’re the ones who have been planning 3,4-diaminopyridine (DAP), a therapy for a rare disorder called Lambert-Eaton Myasthenic Syndrome which is currently being provided at no cost, and run it through the FDA’s regulatory process so as to get market exclusivity for it. The plan then? Charge a lot for it, naturally. Insurance companies will cough up, because there aren’t that many LEMS patients and it’s not worth fighting about, and Catalyst would be able to sit on several years worth of very nice profits with very little risk at all. You may recognize this business plan.
I’ve been blaming the FDA for providing incentives to do this sort of thing, so this latest news is interesting. The agency has sent Catalyst a rare refusal-to-file letter for their drug, saying that the application was incomplete. Further details are lacking, but this is potentially an interesting development.
The press release from Catalyst, by the way, is intensely irritating. They describe themselves as “a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating diseases“, but you’ll note that the word “discovering” is not being used, which is good, because they certain didn’t discover DAP as a therapy for this disease. (Here’s a paper from 1989 on the subject, and that one is referencing even earlier studies. No, “commercializing” is the key word, for sure. And the CEO is quoted as saying “We remain focused on delivering on our promise to transform the way people living with LEMS and CMS are provided access to a safe and effective, FDA approved therapy“. My own take on this is that they’re focused on becoming the only people who can say that they took this old therapy and got the FDA to paste an “approved” label on it, thus transforming the way LEMS patients get access by sending them a nice stiff bill for it instead. The DAP patients are getting now, for free, is most unlikely to be any less “safe and effective” than what Catalyst wants to sell them.
But it’s true, Catalyst has promised to transform the way these patients get access, and they’re good for it, too – in fact, I’m sure that they can hardly wait to start transforming things good and hard. The press release, to my eyes, would be greatly improved by the insertion of the phrase “a drug that someone else is willing to provide at no cost” after every mention of the compound. But they seem to have left that part out.