Evinacumab: First Approval

Drugs. 2021 Jun;81(9):1101-1105. doi: 10.1007/s40265-021-01516-y. Epub 2021 May 18.


The recombinant human monoclonal antibody evinacumab (evinacumab-dgnb, EVKEEZA™) is an angiopoietin-like protein three (ANGPTL3) inhibitor that has been developed by Regeneron Pharmaceuticals for the treatment of homozygous familial hypercholesterolaemia (HoFH), refractory hypercholesterolemia (both familial and non-familial) and severe hypertriglyceridaemia. Based on the results of the phase III ELIPSE HoFH trial, evinacumab was recently approved in the USA as an adjunct to other LDL-C lowering therapies for the treatment of adult and paediatric patients aged 12 years and older with HoFH, and has received a positive opinion in the EU. This article summarizes the milestones in the development of evinacumab leading to this first approval for HoFH.

Publication types

  • Review

MeSH terms

  • Angiopoietin-like Proteins / antagonists & inhibitors
  • Antibodies, Monoclonal / adverse effects
  • Antibodies, Monoclonal / pharmacology
  • Antibodies, Monoclonal / therapeutic use*
  • Anticholesteremic Agents / adverse effects
  • Anticholesteremic Agents / pharmacology
  • Anticholesteremic Agents / therapeutic use*
  • Double-Blind Method
  • Drug Approval
  • Humans
  • Hypercholesterolemia / drug therapy*
  • Hypertriglyceridemia / drug therapy*
  • Multicenter Studies as Topic
  • Randomized Controlled Trials as Topic
  • United States


  • Angiopoietin-like Proteins
  • Antibodies, Monoclonal
  • Anticholesteremic Agents
  • evinacumab