Lomitapide: a review of its clinical use, efficacy, and tolerability

Abstract

Lomitapide is an inhibitor of MTP, an enzyme located in the endoplasmic reticulum of hepatocytes and enterocytes. This enzyme is responsible for the synthesis of very low-density lipoproteins in the liver and chylomicrons in the intestine. Lomitapide has been approved by the US Food and Drug Administration, European Medicines Agency, and other regulatory agencies for the treatment of hypercholesterolemia in adult patients with homozygous familial hypercholesterolemia. Clinical trials have shown that lomitapide reduces low-density-lipoprotein cholesterol levels by around 40% in homozygous familial hypercholesterolemia patients on treatment with statins with or without low-density-lipoprotein apheresis, with an acceptable safety and tolerance profile. The most common adverse events are gastrointestinal symptoms that decrease in frequency with long-term treatment, and the increase in liver fat remains stable. This review analyzes the clinical use, efficacy, and tolerability of lomitapide.

Keywords:

• lomitapide
• homozygous familial hypercholesterolemia
• safety
• efficacy
• cardiovascular disease

Disclosure

PM is the president of the Spanish Familial Hypercholesterolemia Foundation. RA is part of the scientific committee of the Spanish Familial Hypercholesterolemia Foundation, has received honorary fees as speaker and participated in advisory boards from Aegerion, Amgen, AstraZeneca, Abbott, Boehringer-Ingelheim, MSD, and Sanofi, and reports nonfinancial support from Tecnofarma and the European Atherosclerosis Society. AC has received honorary fees as speaker and participated in advisory boards from Abbott, Novo Nordisk, MSD, and Saval. The authors report no other conflicts of interest in this work.