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

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 42 months) was 9.8 mg/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 Lomitapide dose was reduced for 39.2% of drug-related AEs, withdrawn temporarily for 12.2%, and discontinued for 1 event (1.4%).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 +/- 172.0 mg/dl (5.8 +/- 4.5 mmol/l) predose to 159.4 +/- 93.0 mg/dl (4.1 +/- 2.4 mmol/l) at 12 months (p = 0.0245). Conclusion: This interim analysis suggests lomitapide is safe and effective in real-world clinical practice in Japan. What is this article about? Lomitapide is a drug used to treat homozygous familial hypercholesterolemia (HoFH), a rare inherited disorder that causes very high cholesterol levels. Because HoFH is rare, only a limited number of patients were enrolled into the clinical trials that showed it was safe and effective, before its approval. Therefore, a study is now underway to evaluate the efficacy and safety of lomitapide when it is used in daily clinical practice in Japan. We have analyzed data for 39 patients who have been enrolled in this study so far.What are the results? We found that although most patients experienced some side effects, only one patient had to discontinue lomitapide. Most side effects could be managed without having to alter lomitapide treatment, or in some cases by reducing the dose or stopping the drug temporarily. We also found that lomitapide reduced cholesterol levels.What do the results mean? The results suggest that lomitapide is generally safe and effective in patients with HoFH being treated in routine clinical practice. The study is ongoing and additional analyses will be performed when a greater number of patients have been treated. Lomitapide was generally safe and effective in patients with homozygous familial hypercholesterolemia being treated in real-world clinical practice in Japan.