I last wrote about the situation with Catalyst Pharmaceuticals here last December when Firdapse, their drug for Lambert-Eaton myasthenic syndrome, was approved by the FDA. If you know the story, though (or follow the links in that post) you’ll see that referring to it as “their drug” is a rather legalistic way of looking at things. Firdapse is 3,4-diaminopyridine, and it’s been used as a therapy for LEMS for many years. It was, in fact, provided free to the (few) patients with the disease by a small company called Jacobus Pharmaceuticals, until Catalyst saw a regulatory opportunity and hopped in with a chance to take an old generic compound and get marketing exclusivity by taking it through the modern FDA process.
Whereupon they announced a price of $375,000 for the treatment. This has caused a great deal of uproar, and it should, but in that December post I laid a good chunk of the blame right on the FDA for allowing this sort of thing to happen by awarding such generous prizes for un-grandfathering the approval of older drugs. I still think Catalyst are slime for doing this, of course, but it’s the FDA that have held the door open for them and asked if they could hold their coat. Thank you Mister Slime, glad you could join us, can I get you anything to drink?
Well, there’s been an interesting twist in the story, as detailed here at STAT‘s Pharmalot column. Jacobus was also working on getting their 3,4-diaminopyridine approved, and that came through this week. This was a pediatric approval, as opposed to use in adult patients – but physicians are free to prescribe the Jacobus drug off-label. And since the Jacobus trial also included adults, it’s not very far off-label at all. One also needs to consider the fact that the compound has been in actual human use since the 1980s, so it’s not like we’re talking some out-on-a-limb experimental therapy here.
The question is what the Jacobus drug will be priced at, and as I write this, no price has been announced. My guess is that it will be substantially less than the Catalyst price, giving insurance companies even more of an incentive to allow the off-label usage. This is apparently what investors think, too, because Catalyst shares fell off a cliff at the market open yesterday. But at the same time, I don’t think that the drug’s going to be given out for free any more, either. And although I think that the FDA has done the right thing here, it’s only after generally doing the wrong thing by rewarding companies too generously to start with, and that reward system is still in place.