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

Authors Alonso R, Cuevas A, Mata P

Received 3 March 2019

Accepted for publication 8 May 2019

Published 1 July 2019 Volume 2019:14 Pages 19—30

DOI https://doi.org/10.2147/CE.S174169

Checked for plagiarism Yes

Review by Single-blind

Peer reviewers approved by Dr Cristina Weinberg

Peer reviewer comments 3

Editor who approved publication: Professor Garry Walsh


Rodrigo Alonso,1,2 Ada Cuevas,1 Pedro Mata2

1Department of Nutrition, Clínica Las Condes, Santiago, Chile; 2Familial Hypercholesterolemia Foundation, Madrid, Spain

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

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