Evkeeza Approved for Pediatric Homozygous Familial Hypercholesterolemia

Credit: Regeneron.
Evinacumab is a fully-human monoclonal antibody that binds to and blocks the function of angiopoietin-like 3 (ANGPTL3).

The Food and Drug Administration (FDA) has approved Evkeeza® (evinacumab-dgnb) as an adjunct to other lipid-lowering therapies to treat children 5 to 11 years of age with homozygous familial hypercholesterolemia (HoFH).

Evinacumab is a fully-human monoclonal antibody that binds to and blocks the function of angiopoietin-like 3 (ANGPTL3). The approval was based on data from a 3-part single-arm, open-label trial (ClinicalTrials.gov Identifier: NCT04233918) that evaluated the efficacy and safety of evinacumab in 14 pediatrics patients (average age, 9 years) with HoFH who were enrolled in the Part B portion of the trial. Patients had an average LDL-C level of 264mg/dL; 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% (95% CI, -69, -28) at week 24. Moreover, significant reductions were observed in other key secondary endpoints including apolipoprotein B (-41%; 95% CI, -59, -24), non-high-density lipoprotein cholesterol (-49%; 95% CI, -68, -30), and total cholesterol (-49%; 95% CI, -65, -33). Reductions in LDL-C were similar across baseline characteristics, including age, sex, limited LDLR activity, concomitant treatment with lipoprotein apheresis, and concomitant background lipid-lowering medications.

The safety profile of evinacumab in patients 5 years of age and older was consistent with that seen in adults and pediatric patients 12 years of age and older. The most common adverse reactions reported were oropharyngeal pain, upper abdominal pain, diarrhea, headache, and nasopharyngitis.

“By adding Evkeeza to standard lipid-lowering therapies in this pivotal trial, children were able to reduce their LDL-C, with the vast majority able to achieve declines of nearly 50%,” said Carissa M. Baker-Smith, MD, MPH, Co-Director of Nemours Cardiac Center Cardiovascular Research and Innovation Program, Director of Nemours Cardiac Center Pediatric Preventive Cardiology, pediatric cardiologist, and a trial investigator. “These are clinically meaningful results that physicians should consider when developing a treatment approach for these young patients.”

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.


  1. FDA approves first-in-class Evkeeza® (evinacumab-dgnb) for young children with ultra-rare form of high cholesterol. News release. Regeneron Pharmaceuticals, Inc. Accessed March 22, 2023. https://www.globenewswire.com/news-release/2023/03/22/2632063/0/en/FDA-Approves-First-in-class-Evkeeza-evinacumab-dgnb-for-Young-Children-with-Ultra-rare-Form-of-High-Cholesterol.html
  2. Package insert. Regeneron Pharmaceuticals, Inc; 2023. Accessed March 22, 2023. https://www.regeneron.com/downloads/evkeeza_pi.pdf