Homozygous familial hypercholesterolemia produces extreme LDL levels from birth, and patients often respond poorly to therapies that depend on a working LDL receptor. Evkeeza (evinacumab) offered a route around that limitation.
Unlike statins, PCSK9 inhibitors, and most other lipid therapies, Evkeeza works independently of LDL receptor activity, which makes it effective even in receptor-negative patients. The intravenous antibody is given every four weeks as add-on therapy and was the first ANGPTL3 inhibitor approved.
Evkeeza has since expanded into younger pediatric age groups, and as a biologic it carries twelve years of regulatory exclusivity from approval with no biosimilar competition. It is the benchmark ANGPTL3 agent for lipid trial planning.