touchDATA POINT
Long-term hepatic safety of lomitapide in homozygous familial hypercholesterolemia
Learning Objectives
After watching this activity, participants should be better able to:
- Understand the novel mechanism of action of lomitapide
- Describe key hepatic safety outcomes of the lomitapide Phase 3 and extension clinical trial and the LOWER registry
- Recall that the hepatic safety of lomitapide remained favourable for up to 9 years
Overview
Lomitapide is a microsomal triglyceride transfer protein inhibitor, indicated as an adjunctive low-density lipoprotein cholesterol (LDL-C) reducing therapy in patients with homozygous familial hypercholesterolemia (HoFH).1,2 Lomitapide has the potential to lead to idiosyncratic hepatotoxicity and increase hepatic steatosis by reducing the export of lipids from hepatocytes, so hepatic safety is of clinical interest.2 Hepatic safety data were aggregated from three sources, including the pivotal Phase 3 and extension phase clinical trial (median 5.1 years follow-up), 8-year data from the Lomitapide Observational Worldwide Evaluation Registry (LOWER), and real-world evidence from an Italian cohort treated with lomitapide for up to 9 years.3 Data from these studies builds evidence for the favourable long-term hepatic safety profile of lomitapide. No clinically significant elevation of hepatic biomarkers was observed, and hepatic stiffness remained normal for up to 9 years of follow-up.3
References
- Cuchel M, Meagher EA, du Toit TH, et al. Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study. Lancet 2013;381(9860):40-6.
- Cuchel M, Bloedon LT, Szapary PO, et al. Inhibition of microsomal triglyceride transfer protein in familial hypercholesterolemia. N Engl J Med 2007;356(2):148-56.
- Larrey D, D'Erasmo L, O'Brien S, Arca M; Italian Working Group on Lomitapide. Long-term hepatic safety of lomitapide in homozygous familial hypercholesterolaemia. Liver Int 2023;43(2):413-23.
Downloadable slide deck resource is available in the Toolkit.
Downloadable slide deck resource is available in the Toolkit.
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