Abstract 12468: Efficacy and Safety of Lomitapide in Japanese Patients With Homozygous Familial Hypercholesterolemia on Concurrent Lipid-Lowering Therapy

• Published in: Circulation (New York, N.Y.) Vol. 132; no. Suppl_3 Suppl 3; p. A12468
• Main Authors: Harada-Shiba, Mariko, Yoshida, Masayuki, Ikewaki, Katsunori, Nohara, Atsushi, Yanagi, Koji, Otsubo, Yoshihiko, Foulds, Pamela, Sumeray, Mark
• Format: Journal Article
• Language: English
• Published: by the American College of Cardiology Foundation and the American Heart Association, Inc 10.11.2015
• ISSN: 0009-7322; 1524-4539
• DOI: 10.1161/circ.132.suppl_3.12468

IntroductionLomitapide is a microsomal triglyceride transfer protein inhibitor indicated as an adjunctive therapy for adults with homozygous familial hypercholesterolemia (HoFH). In a previous global Phase 3 study in non-Japanese patients with HoFH, lomitapide in combination with other lipid-lowering therapy resulted in a 50% reduction in LDL-C levels from baseline after 26 weeks of treatment. This open-label, multicenter, phase 3 study evaluated the efficacy and safety of lomitapide in adult Japanese patients with HoFH.MethodsAdult patients with HoFH added lomitapide to their maximally tolerated, stable lipid-lowering therapy. Lomitapide was initiated at 5 mg/day and escalated to each patient’s maximum tolerated dose (≤60mg/day) over 14 weeks. The primary endpoint was the mean % change from baseline in LDL-C at Week 26.ResultsNine patients (5 men, 4 women, mean age 50 [33-74] years) received lomitapide (mean dose 20 mg); 8 patients completed 26 weeks of treatment. Mean LDL-C was reduced from 199.3 mg/dL (95% CI148.6-250.0) at baseline to 117.9 mg/dL (95% CI70.2-165.6, last observation carried forward) at Week 26. Mean % LDL-C reduction was significant in patients with and without apheresis; significant reductions were also seen in key secondary endpoints (Table). A >50% reduction in LDL from baseline was achieved by 5/9 (56%) patients. Gastrointestinal symptoms were the most common adverse event (diarrhea in 7/9 [78%] patients). Abnormal liver function tests were reported in 3/9 (33%) patients; 1 patient discontinued treatment after 22 weeks due to persistent liver enzyme elevations. There were no serious adverse events.ConclusionsThe efficacy and safety of 26 weeks of lomitapide in Japanese patients were consistent with results in the global phase 3 study, including a significant reduction in LDL-C and no new safety signals. Treatment in the safety phase (Weeks 26-56) is ongoing.