3,000 in the USA and 15,000 HoFH patients worldwide. Good to know the total number. That's a HUGE market at $295K per patient per year.
Also and this is just my opinion for the long run. Let's say you are heterozygous with difficult to treat LDL that is dangerously high. You are already maxed out on statins etc. Is it not possible that lopitamide could be used selectively in this pt population? Or ANYONE with intractable cholesterol levels. They would need to do the studies of course. The issue with giving the drug to a broader population has always been liver tox which can be monitored and should reverse if the offending agent is discontinued. Once the cardiologists get comfortable prescribing this drug, it is possible that it could be given to a broader population albeit at a discounted price.
Censored argues that CNBC is crooked (well, OK, we'll grant him that one) but this wasn't CNBC reporting, it was an interview with the CEO. The CEO said it and he has a fiduciary responsibility as a corporate officer and can be held liable if he lied. There is no reason for him to lie, he could have made just vague references. He has data to back it up so he is able to state it as fact. Here's a clue - it ain't wikileaks either.