Introduction: Homozygous Familial Hypercholesterolemia (HoFH) is a very severe 
genetic form of hypercholesterolemia. Lacking LDL receptors in the liver, 
subjects with HoFH have raised plasma levels of LDL cholesterol, and up to 100 
times higher risk of premature atherosclerotic cardiovascular disease than the 
general population.Areas covered: This evaluation is of a phase 3 trial of 
evinacumab; Evinacumab Lipid Studies in Patients with Homozygous Familial 
Hypercholesterolemia (ELIPSE HoFH). Evinacumab is a human monoclonal antibody 
inhibitor of angiopoietin-like protein 3 (ANGPTL3). In ELIPSE HoFH, evinacumab 
reduced LDL cholesterol by 47.1 ± 4.6%, HDL cholesterol by 30.4%, and 
triglycerides by 50.4 ± 7.7%.Expert opinion: Evinacumab is not the ideal 
treatment for HoFH as it does not reduce LDL cholesterol levels to treatment 
targets while increasing HDL cholesterol. Although the incidence of adverse 
effects with evinacumab was low in ELIPSE HoFH, further studies are necessary to 
clarify its effects on liver enzymes and clinical cardiovascular outcomes. 
Evinacumab is a candidate to become the standard treatment for HoFH, as it may 
be better tolerated and/or more efficacious than the presently available 
specific treatment (lomitapide). However, the widespread use of evinacumab to 
treat high triglycerides or LDL cholesterol is unlikely due to evinacumab 
decreasing HDL cholesterol.