Positive results in Phase III study of lomitapide (Aegerion Pharmaceuticals) for Homozygous Familial Hypercholesterolemia

New results from a Phase III clinical trial of lomitapide, from Aegerion Pharmaceuticals, show it substantially and stably reduced LDL cholesterol in patients with the orphan disease Homozygous Familial Hypercholesterolemia (HoFH). Twenty-nine adult HoFH patients from across the world were enrolled, with 23 patients completing both the efficacy and the safety phases. All of the patients received lomitapide along with conventional lipid-lowering therapies including statins and, in some cases, apheresis. The lomitapide dose was gradually increased from five mg to a maximum tolerated dose of up to 60 mg per day.

At the end of the efficacy phase, LDL-C levels were reduced by an average of 50 percent from baseline. Approximately one-third of the patients experienced levels of LDL-C that were less than 100 mg/dl � close to the recommended therapeutic goals� at some point during the study, and concomitant lipid-lowering therapy was modified in a subset of these patients during the safety phase. Despite these changes in treatment, patients' mean LDL-C levels were still reduced by 38 percent at the end of the study. Results are published in The Lancet: "Efficacy and safety of a microsomal triglyceride transfer protein inhibitor in patients with homozygous familial hypercholesterolaemia: a single-arm, open-label, phase 3 study" Marina Cuchel et al. The Lancet, Early Online Publication, 2 November 2012 doi:10.1016/S0140-6736(12)61731-0