Evinacumab Cuts LDL-C in Children With Homozygous Familial Hypercholesterolemia

Evinacumab is a fully-human monoclonal antibody that binds to and blocks the function of angiopoietin-like 3

Evinacumab significantly reduced low-density lipoprotein cholesterol (LDL-C) levels in children 5 to 11 years of age with homozygous familial hypercholesterolemia (HoFH), according to findings from a phase 3 trial. 

Evinacumab is a fully-human monoclonal antibody that binds to and blocks the function of angiopoietin-like 3 (ANGPTL3).The single-arm, open-label study (ClinicalTrials.gov Identifier: NCT04233918) evaluated the efficacy and safety of evinacumab in 14 pediatrics patients (average age, 9 years) with an average LDL-C level of 264mg/dL. Among these participants, 86% were on statins, 93% were on ezetimibe, 50% were on LDL apheresis and 14% were on lomitapide

Patients received evinacumab 15mg/kg intravenously every 4 weeks in addition to lipid-lowering therapies for 24 weeks. The primary endpoint was the change in LDL-C from baseline to week 24. Key secondary endpoints included the effect of evinacumab on other lipid parameters.

Results showed evinacumab met the primary endpoint demonstrating a mean reduction in LDL-C of 48% at week 24. Treatment with evinacumab reduced LDL-C by at least half in 79% of patients and was associated with an absolute mean reduction in LDL-C of 132mg/dL.

Reductions were also observed in levels of all lipid endpoint parameters (apolipoprotein B, non-high-density lipoprotein cholesterol, lipoprotein(a), and total cholesterol), generally within the first 8 weeks of treatment. The most common adverse reactions reported were oropharyngeal pain, upper abdominal pain, diarrhea, headache, and nasopharyngitis.

“This latest phase 3 trial illustrates the potential of this medicine to be a breakthrough HoFH therapy for children as young as 5 years old, helping them control their LDL-C early in the course of their disease,” said trial investigator M. Doortje Reijman, MD, Research Associate in Pediatric Metabolic Diseases and Nephrology at the Amsterdam University Medical Center.

Evinacumab is currently marketed under the brand name Evkeeza® and is indicated as an adjunct to other LDL-C lowering therapies for the treatment of patients 12 years of age and older with HoFH. Regeneron plans to submit an application to the Food and Drug Administration (FDA) for the pediatric indication by the end of 2022.


Evkeeza® (evinacumab) phase 3 trial demonstrates 48% LDL-C reduction in children with ultra-rare form of high cholesterol. News release. Regeneron Pharmaceuticals, Inc. Accessed May 21, 2022. https://www.prnewswire.com/news-releases/evkeeza-evinacumab-phase-3-trial-demonstrates-48-ldl-c-reduction-in-children-with-ultra-rare-form-of-high-cholesterol-301552371.html