Real-world safety and efficacy of lomitapide in homozygous familial hypercholesterolemia: interim report of special-use survey in Japan.

Abstract

Aim: To evaluate the safety and efficacy of lomitapide in real-world clinical practice in Japan. Patients & methods: Interim analysis of 39 patients with homozygous familial hypercholesterolemia from an all-case surveillance study. Results: Median lomitapide dose (across 42months) was 9.8mg/day. 74 drug-related adverse events (AEs) were reported in 24 (61.5%) patients, including 14 (35.9%) with liver-related AEs, 19 (48.7%) with gastrointestinal disorders and 1 (2.6%) bleeding disorder. Lomitapide dose was reduced for 39.2% of drug-related AEs, withdrawn temporarily for 12.2%, and discontinued for 1 event (1.4%). Mean±SD blood LDL-C level decreased from 225.9±172.0mg/dl (5.8±4.5mmol/l) predose to 159.4±93.0mg/dl (4.1±2.4mmol/l) at 12months (p=0.0245). Conclusion: This interim analysis suggests lomitapide is safe and effective in real-world clinical practice in Japan.