Homozygous familial hypercholesterolemia is a cardiovascular condition that makes it difficult for the body to remove LDL cholesterol (bad cholesterol) from the blood. Evinacumab is a monoclonal antibody used for the treatment of dyslipidemia, a condition characterized by an abnormal amount of lipids in the blood. This study aims to evaluate the safety and efficacy of evinacumab in the treatment of homozygous familial hypercholesterolemia.

This double-blind, placebo-controlled, phase-3 trial included a total of 65 patients with homozygous familial hypercholesterolemia. The patients were randomly assigned in a 2:1 ratio to receive an intravenous infusion of evinacumab or placebo. The main endpoint was the percent change from baseline (255.1 mg per dL) in the LDL cholesterol levels.

During the trial duration of 24 weeks, patients in the evinacumab group had a 47.1% relative reduction of LD cholesterol levels from the baseline, as compared with 1.9% in the placebo group. The LDL cholesterol level was lower in the evinacumab group in both null-null and non-null patients, as compared with the placebo groups.

The research concluded that the use of evinacumab resulted in a significant reduction in LDL cholesterol levels from the baseline in patients with homozygous familial hypercholesterolemia.

Ref: https://www.nejm.org/doi/full/10.1056/NEJMoa2004215