Background Lomitapide is a lipid-lowering agent indicated as adjunct therapy for adult HoFH Objectives This study evaluated the medium-term effectiveness and safety of lomitapide in a large cohort of HoFH patients in Europe. Methods In a multicenter retrospective, observational study including 75 HoFH patients treated with lomitapide in a real-world clinical setting from 9 European countries, LDL-C changes, adverse events (AEs) as well as major adverse cardiovascular events (MACE) were assessed. Results After a median 19 months (IQR 11-41 months) of treatment with a mean dosage of 20 mg of lomitapide. LDL-C decreased by 60%, from baseline 280.5 mg/dL (191.8-405.0 mg/dl) to 121.6 mg/dl (61.0-190.5 mg/dl). At the last visit, 32.0% of patients achieved LDL-C < 100mg/dL and 18.7% <70 mg/dL. At baseline, 38 HoFH patients were receiving LDL apheresis (LA), but after initiation of lomitapide 36.8% of patients discontinued LA. During follow-up, lomitapide was permanently interrupted in 13% of patients. Gastrointestinal (GI) AEs occurred in 40% and liver transaminases increased (3-5 x ULN) in 13% of patients. Among patients with liver ultrasound evaluation (n = 45), a modest increase in hepatic steatosis was noted during treatment; however liver stiffness measured by elastography in 30 of them remained within the normal range. Among HoFH patients exposed to lomitapide for at least 2 years, MACE incident rate was 7.4 per 1000 person-years in the 2 years after as compared to 21.2 per 1000 person-years before treatment with lomitapide. Conclusions In this medium-term real-world experience, lomitapide proved to be very effective in reducing LDL-C in HoFH. GI AEs were common, but liver safety was reassuring with no sign of increased risk of liver fibrosis. A signal of cardiovascular protection was also observed.

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