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更新于：2025-08-27
Lomitapide Mesylate
甲磺酸洛美他派
更新于：2025-08-27
概要
基本信息
药物类型 小分子化药 |
别名 Lojuxta、lomitapide、Lomitapide Mesilate + [7] |
靶点 |
作用方式 抑制剂 |
作用机制 MTTP抑制剂(微粒体甘油三酯转运蛋白复合体抑制剂) |
非在研适应症- |
最高研发阶段批准上市 |
首次获批日期 美国 (2012-12-21), |
最高研发阶段(中国)申请上市 |
特殊审评孤儿药 (美国)、孤儿药 (欧盟)、临床急需境外新药 (中国) |
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结构/序列
分子式C40H41F6N3O5S |
InChIKeyQKVKOFVWUHNEBX-UHFFFAOYSA-N |
CAS号202914-84-9 |
关联
21
项与 甲磺酸洛美他派 相关的临床试验NCT04681170
Phase III, Single Arm, Open Label, International, Multi Centre Study to Evaluate the Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH) on Stable Lipid Lowering Therapy
EUCTR2018-002911-80-IT
Open-label study to evaluate the safety, tolerability, and efficacy of LOmitapide for the treatment of patients with Familial CHylomicroNEmia Syndrome - LOCHNES
NCT02765841
A Phase 3, Single-arm, Open-label, International, Multi-center Study to Evaluate the Efficacy and Safety of Lomitapide in Pediatric Patients With Homozygous Familial Hypercholesterolemia on Stable Lipid-lowering Therapy
100 项与 甲磺酸洛美他派 相关的临床结果
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100 项与 甲磺酸洛美他派 相关的转化医学
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100 项与 甲磺酸洛美他派 相关的专利（医药）
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333
项与 甲磺酸洛美他派 相关的文献（医药）2025-07-18Cell Death Discovery
Role of S-palmitoylation in digestive system diseases.
Review
作者: Yu, Tao ; Wang, Shuangshuang ; Yuan, Qiuxiang ; Li, Hanqing ; Qi, Xingsi
Digestive system diseases, including liver diseases, gastrointestinal cancers, and inflammatory bowel diseases, pose major health challenges worldwide. These conditions are influenced by a range of key metabolic signaling pathways, many of which are regulated by palmitoylation. Palmitoylation is a type of lipid modification catalyzed by DHHC palmitoyl S-acyltransferases (DHHC-PTAs) and depalmitoylases, which play critical roles in modulating protein localization, stability, and signal transduction. Dysregulation of S-palmitoylation is closely associated with numerous diseases, including these of the digestive system, through multiple key processes such as immune responses, lipid metabolism, and cellular signaling. Decades of investigations have driven the development of a large body of inhibitors targeting zDHHCs and depalmitoylases, such as S-(2-acetamidoethyl) 2-bromohexadecanethioate (MY-D-4), Artemisinin and Lomitapide. This review provides a comprehensive summary of the role of palmitoylation in digestive system diseases, discusses its effect on disease mechanisms. By elucidating the regulatory functions of palmitoylation under these conditions, this review aimed to identify new strategies for the diagnosis and treatment of digestive system disorders.
2025-07-15JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM
Homozygous Familial Hypercholesterolemia in Spain: Data From Registry of the Spanish Atherosclerosis Society
Article
作者: Sánchez-Sobrino, Paula ; Martínez-Hervás, Sergio ; Masana, Luis ; Martín Ordiales, María Mercedes ; Pérez, Antonio ; Blanco-Echevarría, Agustín ; Civeira, Fernando ; Cortés Rodríguez, Begoña ; González-Bustos, Pablo ; Argüeso Armesto, Rosa ; Ibarretxe, Daiana ; Rodríguez-Carnero, Gemma ; Sánchez-Hernández, Rosa M ; Fuentes Jiménez, Francisco
Abstract:
Context:
Homozygous familial hypercholesterolemia (HoFH) is a rare disease characterized by the presence of 2 pathogenic variants in the LDLR, APOB, PCSK9, or LDLRAP1 genes, which cause very high levels of LDL-cholesterol and premature atherosclerotic cardiovascular disease (ASCVD).
Objective:
To analyze the current situation regarding diagnosis, cardiovascular disease, lipid-lowering treatment, and degree of control of lipids in patients with HoFH in the National Dyslipidemia Registry of the Spanish Atherosclerosis Society.
Methods:
Subjects with HoFH, confirmed by the presence of 2 pathogenic variants in the genes mentioned above, included in the registry from 2013 to June 2023 with an updated review were analyzed.
Results:
Of 71 included subjects with HoFH, 40.8% were women, aged 52 [24-62] years, 57 adults and 13 children. The median follow-up was 7 [4-13] years. Age of diagnosis was 14 [2-26] years, with 10% of ASCVD at diagnosis and 27% of current ASCVD at 40.6 (13.4) years of age; 38% were on PCSK9 inhibitors, 9 patients on lomitapide, 9 on LDL apheresis, and 1 patient on evinacumab. Subjects with more than 4 therapies achieved >80% reduction in LDLc. In the last visit, the median LDLc was 139.3 [89.4-204.2] mg/dL. ASCVD was strongly associated with male sex and family history of ASCVD, relative risk 5.26 (1.53-18.10) and 2.53 (1.03-6.26), P < .05, respectively. Only 18% and 10% meet the recommended LDLc goal in primary and secondary prevention respectively.
Conclusion:
The current situation of HoFH in Spain is better than expected, with marked reductions in LDLc levels with new treatments. In this population, recommended LDLc goals are difficult to achieve despite maximum lipid-lowering therapy. ASCVD has been reduced and delayed by 2 decades.
2025-07-01ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY
Real-World Effectiveness and Safety of Evinacumab in Children and Adults With Homozygous Familial Hypercholesterolemia: A Multisite US Perspective—Brief Report
Article
作者: Malloy, Mary J. ; Warden, Bruce A. ; Shah, Prediman K. ; Horan, Mary ; Wilkinson, Michael J. ; Newfield, Ron S. ; Bijlani, Priyesha ; Ito, Matthew K. ; Stock, Eveline O. ; Davidson, Michael H. ; Pradeep, Pallavi ; Duell, P. Barton
BACKGROUND::
Homozygous familial hypercholesterolemia (HoFH) is an autosomal semidominant disorder characterized by extreme elevations in LDL-C (low-density lipoprotein cholesterol) and early-onset atherosclerotic cardiovascular disease. Evinacumab is a monoclonal antibody administered by monthly intravenous infusion that binds ANGPTL3 (angiopoietin-like 3) and when added to standard lipid-lowering therapies lowers LDL-C by ≈50% in HoFH clinical trials. Studies examining the real-world effectiveness and safety of evinacumab are limited.
METHODS::
We performed a retrospective study to assess the effectiveness and safety of evinacumab in patients with HoFH in clinical practice at 6 US academic medical centers. The primary end point was the percent change in LDL-C from baseline to first follow-up and to the most recent follow-up after evinacumab initiation. Secondary end points were percent change in non–high-density lipoprotein cholesterol (non–HDL-C), triglycerides, total cholesterol, HDL-C, and achievement of an LDL-C <70 mg/dL. Adverse events were recorded.
RESULTS::
Twenty-four patients (mean age, 40 [range, 5–84] years) with HoFH were followed for a median of 48 weeks. Fifty percent were female, 66.7% had atherosclerotic cardiovascular disease, 87.5% were on a statin, 83.3% were on ezetimibe, 66.7% were on PCSK9i (proprotein convertase subtilisin/kexin type 9 inhibitors), 24% were on lomitapide, and 33.3% were undergoing lipoprotein apheresis. Significant reductions in LDL-C, non–HDL-C, total cholesterol, triglycerides, and HDL-C were observed both at the first follow-up (4 weeks) and the most recent follow-up (48 weeks); mean±SEM percent change from baseline to the most recent follow-up was as follows: LDL-C, −53.2% (±4.1); non–HDL-C, −52.7% (±3.9); triglycerides, −47.4% (±5.1); total cholesterol, −48.9% (±4.0); and HDL-C, −30.2% (±4.1);
P
<0.001 for all. Significantly more patients achieved LDL-C <70 mg/dL after evinacumab was added. Nine (37.5%) patients reported adverse events during or following evinacumab infusions. Treatment was discontinued by 1 patient because of back pain.
CONCLUSIONS::
Across 6 US academic medical centers, evinacumab was generally well tolerated by patients with HoFH and lowered LDL-C by ≈50%, consistent with results from clinical trials.
100 项与 甲磺酸洛美他派 相关的药物交易
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研发状态
批准上市
10 条最早获批的记录, 后查看更多信息
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| II a型高脂蛋白血症 | 墨西哥 | 2014-02-09 | |
| 纯合子家族性高胆固醇血症 | 美国 | 2012-12-21 |
未上市
10 条进展最快的记录, 后查看更多信息
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| II型高脂蛋白血症 | 临床3期 | 美国 | 2009-10-29 | |
| II型高脂蛋白血症 | 临床3期 | 加拿大 | 2009-10-29 | |
| II型高脂蛋白血症 | 临床3期 | 意大利 | 2009-10-29 | |
| II型高脂蛋白血症 | 临床3期 | 南非 | 2009-10-29 | |
| 高脂血症 | 临床2期 | 美国 | 2007-10-01 | |
| 高胆固醇血症 | 临床2期 | 美国 | 2006-05-01 | |
| 结直肠癌 | 临床前 | 韩国 | 2022-12-08 |
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临床结果
临床结果
适应症
分期
评价
查看全部结果
临床3期 | 18 | 壓淵網膚餘廠鏇積觸衊(糧餘遞觸積鏇顧餘構夢) = 襯鑰積醖顧範憲獵網餘 鑰鹹餘獵鹽鏇構廠糧壓 (願遞艱襯齋鹽選鑰鏇願 ) | 积极 | 2022-09-10 | |||
临床3期 | 19 | 艱衊範壓鹹鹽壓齋獵壓(構鹹夢蓋顧構鏇鬱衊鏇) = 鑰糧鏇獵積淵製窪鑰遞 範鏇糧獵餘網廠網鹽遞 (鹹選醖鹹廠膚廠積壓鑰, 31.35) 更多 | - | 2018-05-11 | |||
临床1期 | - | 32 | (Lomitapide Sprinkled in Applesauce (Treatment A)) | 醖窪鑰憲願夢築製鑰鹹(糧繭齋遞觸網糧顧鬱製) = 鹽遞鏇襯壓糧壓艱襯選 範觸鏇範積艱繭鹹網構 (壓遞衊鏇鏇構顧積獵蓋, 15.2) 更多 | - | 2018-02-13 | |
(Lomitapide Sprinkled in Mashed Banana (Treatment B)) | 醖窪鑰憲願夢築製鑰鹹(糧繭齋遞觸網糧顧鬱製) = 艱衊遞遞選窪簾齋鑰遞 範觸鏇範積艱繭鹹網構 (壓遞衊鏇鏇構顧積獵蓋, 13.4) 更多 | ||||||
临床3期 | 29 | 網鏇遞艱淵蓋廠憲築醖(憲鹽壓製鬱醖鑰獵鑰夢) = 蓋顧膚糧獵淵淵憲鑰淵 衊鹽觸鑰醖構製壓齋鹽 (鬱遞齋艱顧醖製範選積 ) | 积极 | 2015-06-01 | |||
(Lipoprotein apheresis) | 網鏇遞艱淵蓋廠憲築醖(憲鹽壓製鬱醖鑰獵鑰夢) = 築糧夢顧積構鹹淵淵膚 衊鹽觸鑰醖構製壓齋鹽 (鬱遞齋艱顧醖製範選積 ) | ||||||
临床2期 | 44 | (Atorvastatin 20 mg) | 觸鹹糧餘觸鬱選製觸糧(醖鏇憲膚顧襯顧選鬱積) = 膚糧廠餘醖願簾艱顧鹹 簾鹹鏇廠鑰鬱夢鬱鹽廠 (鏇淵顧範網範廠鏇艱膚, 14.35) 更多 | - | 2013-02-25 | ||
(Atorvastatin 20 mg + Lomitapide) | 觸鹹糧餘觸鬱選製觸糧(醖鏇憲膚顧襯顧選鬱積) = 鹹醖淵糧齋願蓋鬱範鹹 簾鹹鏇廠鑰鬱夢鬱鹽廠 (鏇淵顧範網範廠鏇艱膚, 26.78) 更多 | ||||||
临床3期 | 29 | 膚鑰願鑰齋構鬱廠艱夢(繭齋憲憲廠築餘選製憲) = 醖顧製繭糧積壓獵積範 鬱壓鹹願獵觸觸齋積齋 (獵鏇壓膚壓鬱遞願窪壓, 31.25) 更多 | - | 2013-02-22 | |||
临床2期 | 6 | 構鹽觸憲膚糧糧膚膚範(繭鬱鏇鹹窪願願製醖餘) = 鏇糧襯網積糧構網壓膚 顧壓遞窪餘鏇繭艱範願 (鑰顧觸繭築遞壓夢壓壓, 9.311) 更多 | - | 2013-02-22 | |||
临床2期 | 260 | placebo (Placebo) | 糧鑰糧蓋廠餘選獵繭衊(鬱觸衊遞蓋選憲齋製膚) = 膚餘廠艱窪餘齋鑰衊簾 選選壓簾醖艱築鹽糧鏇 (繭製獵鹽蓋糧鏇願廠蓋, 1.814) 更多 | - | 2013-02-22 | ||
(AEGR-733 5 mg) | 糧鑰糧蓋廠餘選獵繭衊(鬱觸衊遞蓋選憲齋製膚) = 壓淵範鬱觸廠窪鏇衊繭 選選壓簾醖艱築鹽糧鏇 (繭製獵鹽蓋糧鏇願廠蓋, 6.297) 更多 | ||||||
临床2期 | 157 | Placebo (Placebo) | 繭窪繭鑰鏇衊顧遞築醖(鹽醖窪構選獵衊餘鬱窪) = 糧願網廠餘選鑰鬱憲鬱 淵淵糧鹽網窪鏇顧構繭 (繭窪夢觸淵鑰襯齋齋遞, 11.2) 更多 | - | 2013-02-21 | ||
(Atorvastatin 20 mg) | 繭窪繭鑰鏇衊顧遞築醖(鹽醖窪構選獵衊餘鬱窪) = 憲遞積膚鏇構簾鹽鹹膚 淵淵糧鹽網窪鏇顧構繭 (繭窪夢觸淵鑰襯齋齋遞, 16.4) 更多 |
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