Background: Lomitapide (Juxtapid® in US and Lojuxta® in Europe) is the first developed inhibitor of the Microsomal Triglyceride Transfer Protein (MTP) approved as a novel drug for the management of Homozygous Familial Hypercholesterolemia (HoFH). It acts by binding directly and selectively to MTP thus decreasing the assembly and secretion of the apo-B containing lipoproteins both in the liver and in the intestine.

Aims: The present review aims at summarizing the recent knowledge on lomitapide in the management of HoFH.

Results: The efficacy and safety of lomitapide have been evaluated in several trials and it has been shown a reduction of the plasma levels of Low-Density Lipoprotein Cholesterol (LDL-C) by an average of more than 50%. Although the most common side effects are gastrointestinal and liver events, lomitapide presents generally with a good tolerability and satisfactory patients compliance. Recently, in Europe, to evaluate the long-term safety and efficacy of lomitapide, the LOWER registry (ClinicalTrials.gov Identifier: NCT02135705) has been established in order to acquire informations on HoFH lomitapidetreated patients from “real life” clinical practice.

Furthermore, the observation that lomitapide decreases triglyceride levels may be considered for patients affected by severe forms of hypertriglyceridemia who undergo recurrent episodes of pancreatitis and are poor responders to conventional treatment.

Conclusion: Lomitapide represents an innovative and efficacious drug for the treatment of HoFH. Longterm safety data, treatment of pediatric and pregnant HoFH patients and management of severe hypertriglyceridemia still require further investigations.

背景：Lomitapide（美国的Juxtapid®和欧洲的Lojuxta®）是第一个开发的微粒体甘油三酸酯转移蛋白（MTP）抑制剂，被批准作为治疗纯合子家族性高胆固醇血症（HoFH）的新药。它通过直接和选择性地与MTP结合而起作用，从而减少了肝脏和肠道中含有载脂蛋白B的脂蛋白的组装和分泌。

目的：本综述旨在总结HoFH管理中有关洛米他肽的最新知识。

结果：已在多项试验中评估了洛米肽的疗效和安全性，结果表明低密度脂蛋白胆固醇（LDL-C）的血浆水平平均降低了50％以上。尽管最常见的副作用是胃肠道和肝脏事件，但洛米肽显示出良好的耐受性和令人满意的患者依从性。最近，在欧洲，为了评估洛米肽的长期安全性和有效性，已建立LOWER注册表（ClinicalTrials.gov标识符：NCT02135705），以便从“现实生活”临床实践中获得有关用HoFH罗米肽治疗的患者的信息。

此外，对于受严重形式的高甘油三酯血症影响且复发性胰腺炎且对常规治疗反应较差的患者，可以考虑使用洛米他肽降低甘油三酯水平的观察结果。

结论：Lomitapide代表一种新颖有效的HoFH药物。长期安全性数据，小儿和孕妇HoFH患者的治疗以及严重高甘油三酯血症的治疗仍需进一步研究。