Trial reportJournal of atherosclerosis and thrombosis2019
Safety and Efficacy of Lomitapide in Japanese Patients with Homozygous Familial Hypercholesterolemia (HoFH): Results from the AEGR-733-301 Long-Term Extension Study.
Trial report in Journal of atherosclerosis and thrombosis, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
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Who cites it
24 citing papers in PubMed.
- Comparison of Clinical Characterization and Therapeutic Strategies between Homozygous Familial Hypercholesterolemia and Heterozygous Familial Hypercholesterolemia.Journal of atherosclerosis and thrombosis · 2026Article
- Lomitapide mesylate and lomitapide target ALDOA to inhibit growth and enhance gemcitabine efficacy in PDAC.iScience · 2026Article
- Diagnostic and Therapeutic Challenges of Homozygous and Severe Heterozygous Familial Hypercholesterolemia from Clinical Aspect-A Single-Center Study.Journal of clinical medicine · 2025Article
- Research advances in current drugs targeting hyperlipidemia (Review).Molecular medicine reports · 2025Review
- Liver Transplantation in a Child With Homozygous Familial Hypercholesterolemia: A Case Report and Literature Review.Reviews in cardiovascular medicine · 2025Review
- Lipoprotein(a)-Lowering Drugs: A Mini Review.Journal of clinical medicine research · 2025Review
- Review
- Japan Atherosclerosis Society (JAS) Guidelines for Prevention of Atherosclerotic Cardiovascular Diseases 2022.Journal of atherosclerosis and thrombosis · 2024Article
- Advances in Treatment of Dyslipidemia.International journal of molecular sciences · 2023Review
- The Role of Triglyceride-rich Lipoproteins and Their Remnants in Atherosclerotic Cardiovascular Disease.European cardiology · 2023Review
- Current Treatment Options in Homozygous Familial Hypercholesterolemia.Pharmaceuticals (Basel, Switzerland) · 2022Review
- Review
- Perioperative Management and Clinical Outcomes of Liver Transplantation for Children with Homozygous Familial Hypercholesterolemia.Medicina (Kaunas, Lithuania) · 2022Article
- Advancements in the Treatment of Homozygous Familial Hypercholesterolemia.Journal of atherosclerosis and thrombosis · 2022Review
- Novel Pharmaceutical and Nutraceutical-Based Approaches for Cardiovascular Diseases Prevention Targeting Atherogenic Small Dense LDL.Pharmaceutics · 2022Review
- Individualized Treatment for Patients With Familial Hypercholesterolemia.Journal of lipid and atherosclerosis · 2022Review
- Homozygous Familial Hypercholesterolemia.Journal of atherosclerosis and thrombosis · 2021Review
- Challenges of Precision Medicine for Atherosclerotic Cardiovascular Disease Based on Human Genome Information.Journal of atherosclerosis and thrombosis · 2021Review
- Novel therapeutic targets and agents for pediatric dyslipidemia.Therapeutic advances in endocrinology and metabolism · 2021Review
- Management of Familial Hypercholesterolemia: Current Status and Future Perspectives.Journal of the Endocrine Society · 2021Review
Corrections and comments
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Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
aimLomitapide is an approved lipid-lowering agent indicated as adjunct to low-fat diet and standard lipid-lowering therapies (LLTs) including lipoprotein apheresis for the treatment of homozygous familial hypercholesterolemia (HoFH). Clinical data from Phase 3 studies have demonstrated the prolonged lipid-lowering capacity of lomitapide in patients with HoFH. We assessed the long-term lipid-lowering capacity of daily oral lomitapide in a cohort of Japanese patients with HoFH enrolled in a Phase 3 extension study.
methodsFive of 8 Japanese HoFH patients completing a 56-week Phase 3 dose-escalation and safety study of lomitapide continued their maximum tolerated dose (MTD) until study drug was approved or commercially available or until treatment was discontinued. Lipid parameters were measured at Day 1 and at 12-week intervals through study end. Safety and tolerability were assessed.
resultsDaily lomitapide treatment with permitted LLTs maintained approximately 50% mean reductions in plasma low-density lipoprotein cholesterol (LDL-C) levels from baseline for ＞60 weeks. Reductions in LDL-C levels varied across patients and were not associated with the HoFH genotype. Four patients achieved ＞25% reductions and 1 patient achieved ＞50% reduction in LDL-C; 2 patients achieved reduction in LDL-C to ＜100 mg/dL. Lomitapide significantly reduced total cholesterol (－26.5%), triglycerides (－54.8%), and non-high-density lipoprotein cholesterol (non-HDL-C) (－37.4%). All 5 patients continued their individual MTD of lomitapide throughout the extension study with acceptable safety and tolerability, and no deaths were reported.
conclusionResults from this extension study support the long-term safety and efficacy of lomitapide in significantly reducing plasma levels of atherosclerotic lipids in patients with HoFH.
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