Physicians' Academy for Cardiovascular Education

Oral microsomal triglyceride transfer protein inhibitor is beneficial in HoFH

Target achievement and cardiovascular event rates with Lomitapide in homozygous Familial Hypercholesterolaemia

Literature - Blom DJ, Cuchel M, Ager M, et al. - Orphanet J Rare Dis 2018 2018;13:96

Introduction and Methods

According to the European Atherosclerosis Society (EAS) recommendations, patients with homozygous familial hypercholesterolemia (HoFH) should be treated to the same LDL-c targets as other hypercholesterolemia patients, which, however, is not realistic in the majority of HoFH patients with currently available standard therapies [1]. Lomitapide, an oral microsomal triglyceride transfer protein inhibitor, has been approved as adjunctive therapy in adult patients with HoFH [2,3].

In this retrospective analysis, the number of HoFH patients receiving lomitapide who reach EAS targets (LDL-c level ≤70 mg/dL) was assessed. For this purpose, data from two lomitapide studies were used: a single-arm, open label, phase 3 clinical trial of lomitapide in adult patients with HoFH [2], and a single-arm extension of the study [3]. In both studies, the lomitapide dose was gradually increased from 5 mg to a maximum of 60 mg/day, and given on top of standard therapy until week 26. The primary endpoint was the mean percent change in LDL-c levels from baseline to week 26, after which patients remained on lomitapide until week 78 for safety assessment.

In addition, CV event rates in patients receiving lomitapide were compared with published data: the publication by Duell et al., including HoFH patients, who received mipomersen therapy for at least 12 months [4] and the publication by Raal et al., including a HoFH cohort treated with evolocumab [5].

MACE was defined as CV death, non-fatal myocardial infarction, coronary revascularization, unstable angina and/or ischemic stroke.

Main results


In patients with HoFH, lomitapide led to a significant reduction of LDL-c levels and to achievement of EAS targets in approximately half of lomitapide-treated patients after at least 2 years on treatment. CV event rates correlated with LDL-c levels.


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