Except as a result of the price tag, they priced themselves out of the market and any possibility of revenue. That is not a rational. And efficacy isn't really in question: Perfect cure or not, it has a vast increase in quality of life including the ability to have children, avoid pancreatitis, and enjoy alcoholic beverages to name a few. If insurance companies are covering replacement therapy to the tune of $300,000k/year, that would seem a pretty good sweet spot pricing that insurance companies would clamor to approve due to the savings over annual costs.
I don’t think they priced themselves out of a market. Uniqure set the list price at $1M. In Europe each country negotiates their own drug price, so the real price would have been even lower than $1M.
I’m going to speculate that the drug just wasn’t that good, so the demand wasn’t there and the program folded.
I know Germany pays close to $1M for a specific hemophilia therapy and they pay that annually. The price itself wasn’t the issue, this it must have been the drug itself.
according to the article, and a few google searches outside of it, the drug was that good. The article's implicit conclusion was that the drug was priced too high, and offers evidence to back that claim. You seem to be assuming a perfect rational market here that has a rational justification, but real markets don't always work that way.
As someone who works in the industry, the article was a 30,000 ft view and didn’t do a good job of digging into the issue.
Regardless, it looks like the drug does reduce pancreatitis attacks, but only by 50%. So it certainly doesn’t “cure” the patient.
The clinical trials were also quite small, so possibly the national payers said “$1M is reasonable if you do X, but you don’t have the data to convince me you can do X.”
