The lipid-lowering effects of lomitapide are unaffected by adjunctive apheresis in patients with homozygous familial hypercholesterolaemia – a post-hoc analysis of a Phase 3, single-arm, open-label trial.

OBJECTIVE: Lomitapide (a microsomal triglyceride transfer protein inhibitor) is an adjunctive treatment for homozygous familial hypercholesterolaemia (HoFH), a rare genetic condition characterised by elevated low-density lipoprotein-cholesterol (LDL-C), and premature, severe, accelerated atherosclerosis. Standard of care for HoFH includes lipid-lowering drugs and lipoprotein apheresis. We conducted a post-hoc analysis to evaluate the interaction between these therapies.

METHODS: The analysis utilised pooled data from participants in a single-arm, open-label Phase 3 trial. The efficacy of lomitapide as measured by percentage change in LDL-C was compared between subgroups receiving or not receiving concurrent long-term lipoprotein apheresis.

RESULTS: The lipid-lowering effects were consistent across subgroups. Adjunctive apheresis did not statistically significantly alter the efficacy of the lomitapide regimen in managing LDL-C levels in the studied cohort.

CONCLUSION: Lomitapide remains an effective therapeutic option for HoFH patients regardless of their concurrent apheresis status, providing robust lipid control in severe genetic hypercholesterolaemia cases.