Efficacy and safety of lomitapide in Japanese patients with homozygous familial hypercholesterolemia

Aim: there is an unmet need in japan for more optimal lipid-lowering therapy (llt) for patients with homozygous familial hypercholesterolemia (hofh) who respond inadequately to available drug therapies and/or apheresis,to achieve goals of low-density lipoprotein cholesterol (ldl-c) reduction by 50% or to <100 mg/dl. methods: in this study,japanese patients with hofh on stable llt and diet were treated with lomitapide,initiated at 5 mg/day and escalated to maximum tolerated dose (up to 60 mg/day) over 14 weeks. the primary efficacy endpoint was mean percentage change from baseline to week 26 in ldl-c. secondary endpoints included changes in other lipid parameters and safety throughout the 56-week study (including follow-up). results: nine patients entered the efficacy phase of the study and,of these,eight completed 56 weeks. mean ldl-c was reduced by 42% (p<0.0001) at 26 weeks,from 199 mg/dl (95% ci: 149–250) at baseline to 118 mg/dl (95% ci: 70–166). a 50% reduction in ldl-c and ldl-c < 100 mg/dl was achieved by five and six of nine patients,respectively,at 26 weeks. after 56 weeks,ldl-c was reduced by 38% (p=0.0032) from baseline. significant reductions in non-hdl-c,vldl-c,triglycerides,and apolipoprotein b were also reported at week 26. there were no new safety signals and,similar to previous studies,gastrointestinal adverse events were the most common adverse events. conclusion: lomitapide,added to ongoing treatment with other llts,was effective in rapidly and significantly reducing the levels of ldl-c and other atherogenic apolipoprotein b-containing lipoproteins in adult japanese patients with hofh. © 2017 japan atherosclerosis society.