Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

Summary

The primary objective of the study is to demonstrate the reduction of low-density lipoprotein cholesterol (LDL-C) by evinacumab intravenously (IV) in comparison to placebo after 24 weeks in patients with homozygous familial hypercholesterolemia (HoFH). The secondary objectives of the study are to evaluate the effect of evinacumab IV on other lipid parameters, evaluate the effect of evinacumab on LDL-C goal attainment, assess the effect of evinacumab on eligibility for apheresis (using German and US apheresis criteria), evaluate the safety and tolerability of evinacumab in patients with HoFH, assess the pharmacokinetics (PK) of evinacumab in patients with HoFH and evaluate the potential development of anti-evinacumab antibodies.

Timeline

Start

2018-01-18

Primary completion

2019-06-10

Completion

2020-03-17

Outcome

Met primary endpoint

registry analysis (superiority test); Placebo IV Q4W vs Evinacumab 15 mg/kg IV Q4W; p < 0.0001; Least Squares (LS) Mean Difference -49.0 NCT03399786 ↗

release “The trial met its primary endpoint, showing that adding evinacumab to other lipid-lowering therapies decreased LDL cholesterol by 49% on average” regeneron.com ↗

Publications

• Iannuzzo G, Calcaterra I, Gentile M, Stanzione C, De Ruberto F, Di Taranto MD, Fortunato G, Di Minno M. Evinacumab for Homozygous Familial Hypercholesterolemia: The Italian Cohort of the ELIPSE HoFH Study. Adv Ther. 2025 May;42(5):2465-2479. doi: 10.1007/s12325-025-03160-4. Epub 2025 Apr 2.
• Raal FJ, Rosenson RS, Reeskamp LF, Hovingh GK, Kastelein JJP, Rubba P, Ali S, Banerjee P, Chan KC, Gipe DA, Khilla N, Pordy R, Weinreich DM, Yancopoulos GD, Zhang Y, Gaudet D; ELIPSE HoFH Investigators. Evinacumab for Homozygous Familial Hypercholesterolemia. N Engl J Med. 2020 Aug 20;383(8):711-720. doi: 10.1056/NEJMoa2004215.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Evinacumab	Monoclonal antibody	15 mg/kg	Intravenous

Indications