Evkeeza Approved for Homozygous Familial Hypercholesterolemia

Credit: Regeneron.
The approval was based on data from the double-blind, placebo-controlled phase 3 ELIPSE HoFH trial.

The Food and Drug Administration (FDA) has approved Evkeeza™ (evinacumab-dgnb; Regeneron) as an adjunct to other low-density lipoprotein cholesterol (LDL-C) lowering therapies in patients 12 years of age and older with homozygous familial hypercholesterolemia (HoFH).

Evkeeza is a fully-human monoclonal antibody that binds to and blocks the function of angiopoietin-like 3 (ANGPTL3) using the Company’s proprietary proprietary VelocImmune® technology. ANGPTL3 acts as an inhibitor of lipoprotein lipase and endothelial lipase, and appears to play a central role in lipoprotein metabolism.

The approval was based on data from the double-blind, placebo-controlled phase 3 ELIPSE HoFH trial (ClinicalTrials.gov: NCT03399786) that evaluated the efficacy and safety of evinacumab in 65 patients 12 years of age and older with HoFH. Patients were randomly assigned 2:1 to receive either evinacumab 15mg/kg intravenously every 4 weeks or placebo. Patients were on the following lipid-lowering therapies: statins, ezetimibe, proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitorslomitapide, and lipoprotein apheresis. The primary end point was the reduction of LDL-C from baseline to week 24.

Results showed that patients treated with evinacumab had a 47% reduction in LDL-C compared with a 2% increase for patients treated with placebo (absolute change in LDL-C from baseline was 132mg/dL; <.0001). Moreover, 47% of patients in the evinacumab arm achieved LDL-C levels of <100mg/dL (average baseline LDL-C level was 260mg/dL) compared with 23% in the placebo arm (nominal P =.0203).

Additionally, evinacumab met key secondary end points including statistically significant reductions in apolipoprotein B, non-high-density lipoprotein cholesterol, total cholesterol, and triglycerides, compared with placebo (P <.0001 for all).

“Evkeeza, through its unique mechanism of action, was shown to reduce LDL-C levels in patients with all forms of HoFH, even those with nearly no LDL receptor activity, and represents a highly meaningful improvement in our ability to control LDL-C levels in patients with HoFH,” said Daniel J. Rader, MD, Professor and Chair of the Department of Genetics in the Perelman School of Medicine of the University of Pennsylvania, a leading HoFH expert who was involved with Evkeeza clinical trials.

As for safety, evinacumab was generally well tolerated with the most common adverse reactions (incidence greater than or equal to 5%) being nasopharyngitis, influenza-like illness, dizziness, rhinorrhea, and nausea.

Evkeeza is supplied as 345mg/2.3mL and 1200mg/8mL single-dose vials. Treatment is administered based on weight once a month via intravenous infusion.

The average wholesale acquisition cost per patient varies based on weight and is expected to be approximately $450,000 per year on average. Regeneron is offering a patient support program called myRARE that provides financial assistance to eligible patients, as well as product information, insurance benefit verification, community resources, and appointment reminders.


1.    FDA approves first-in-class Evkeeza™ (evinacumab-dgnb) for patients with ultra-rare inherited form of high cholesterol. [press release]. Tarrytown, NY: Regeneron Pharmaceuticals; February 11, 2021. 

2.    Evkeeza™. [package insert]. Tarrytown, NY: Regeneron Pharmaceuticals, Inc.; 2021.