Robyn Karnauskas at Deutsche Bank issued a note saying that because Amgen’s PCSK9 shot lowered LDL (low density lipoprotein) cholesterol by only 19%, compared to 50% for Juxtapid, that Amgen’s drug would not be competitive with Aegerion’s.
Further, it wasn’t clear that Amgen’s PCSK9 drug would even work in the patients with the rare genetic disease that Juxtapid treats, called homozygous familial hypercholesterolemia or HoFH . That’s because blocking PCSK9 lowers cholesterol by blocking turning up the LDL cell receptor, which sucks cholesterol out of the blood. HoFH patients have high cholesterol because their LDL receptors don’t work. So it was unclear whether a PCSK9 drug would do anything for HoFH patients.
So the Amgen drug may not do anything whatsoever for HoFH patients.
Kynmaro is even less effective and has liver toxicity issues that make it unsafe.
Game, Set, Match: Juxtapid (lomitapide)