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Evinacumab for the treatment of homozygous familial hypercholesterolemia

Yanli Gao1, Baoqi Zhang2, Junyi Yang1

  • 1Department of Clinical Pharmacy, Linyi Central Hospital, Linyi, Shandong, China.

Expert Review of Clinical Pharmacology
|February 28, 2022
PubMed

Insights

Evinacumab, an ANGPTL3 inhibitor, significantly reduced LDL-C by 47% in patients with homozygous familial hypercholesterolemia (HoFH). This monoclonal antibody demonstrates potential as a valuable treatment for HoFH, showing good tolerability in clinical trials.

Area of Science:

  • Cardiovascular Medicine
  • Pharmacology

Background:

  • Hypercholesterolemia, linked to abnormal lipoprotein metabolism, elevates cardiovascular disease risk.
  • Angiopoietin-like protein 3 (ANGPTL3) elevates LDL-C by inhibiting lipoprotein lipase.
  • Evinacumab targets ANGPTL3, reducing LDL-C and showing promise for homozygous familial hypercholesterolemia (HoFH).

Purpose of the Study:

  • To review the pharmacological characteristics, clinical evidence, and safety of evinacumab.
  • To evaluate evinacumab's efficacy in patients with homozygous familial hypercholesterolemia (HoFH).

Main Methods:

  • Comprehensive literature search of PubMed (Jan 2000-Aug 2021) using keywords ANGPTL3, evinacumab, and HoFH.
  • Inclusion of Phase 1, 2, and 3 clinical trials, product labeling, and ClinicalTrials.gov data.
  • Review of pharmacological properties, clinical efficacy, and safety data for evinacumab.

Main Results:

  • Phase 3 trials demonstrated evinacumab reduced LDL-C by 47% in HoFH patients, versus a 2% increase with placebo.
  • Evinacumab was generally well-tolerated.
  • Common adverse events included nasopharyngitis, influenza-like illness, dizziness, rhinorrhea, and nausea.

Conclusions:

  • Evinacumab is an effective ANGPTL3 inhibitor for treating HoFH.
  • The drug exhibits a favorable safety profile, with manageable adverse events.
  • Evinacumab presents a significant potential as a therapeutic option for HoFH patients.
Abstract

Keywords:
Low-density lipoprotein cholesterolangiopoietin-like 3evinacumabhomozygous familial hypercholesterolemiahypertriglyceridemia

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