Comparable efficacy of lomitapide in men and women, but side effects differ in HoFH

Lomitapide effectively reduces LDL-C levels in men and women with homozygous familial hypercholesterolemia (HoFH), but women may experience greater LDL-C reductions at specific time points and have a higher prevalence of gastrointestinal side effects compared to men, according to a study.

This post-hoc analysis from the Pan-European Lomitapide Study included 75 patients with HoFH (38 women and 37 men) receiving lomitapide, a microsomal triglyceride transfer protein inhibitor, across Europe. Baseline characteristics and cardiovascular risk factors were similar between sexes. Although overall LDL-C reductions were comparable, women showed a trend toward greater LDL-C reductions at 6 months (-53.0% vs -32.9%; P = 0.051). However, long-term reductions did not differ significantly.

Adverse events, primarily gastrointestinal disturbances, were more frequent in women than men (78 vs 32 events; P = 0.0002), though these side effects were generally manageable. Importantly, event-free survival for ASCVD did not significantly differ between sexes.

Reference
Pavanello C, Suppressa P, Castiglione S, et al; Italian and European Working Group on Lomitapide in HoFH. Sex-related differences in response to lomitapide in HoFH: A subanalysis of the Pan-European Lomitapide retrospective observational study. Atherosclerosis. 2024;401:119089. doi: 10.1016/j.atherosclerosis.2024.119089. Epub ahead of print. PMID: 39708636.