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更新于：2026-09-04
Maralixibat Chloride
氯马昔巴特
更新于：2026-09-04
概要
基本信息
药物类型 小分子化药 |
别名 Lopixibat、Lopixibat chloride、Lum-001 cation + [14] |
靶点 |
作用方式 抑制剂 |
作用机制 ISBT抑制剂(回肠胆汁酸转运体抑制剂) |
在研适应症 |
非在研适应症 |
最高研发阶段批准上市 |
首次获批日期 美国 (2021-09-29), |
最高研发阶段(中国)批准上市 |
特殊审评孤儿药 (美国)、孤儿药 (欧盟)、孤儿药 (中国台湾)、孤儿药 (日本)、孤儿药 (韩国)、优先审评 (中国)、突破性疗法 (美国) |
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结构/序列
分子式C40H56ClN3O4S |
InChIKeyPOMVPJBWDDJCMP-RUKDTIIFSA-M |
CAS号228113-66-4 |
关联
26
项与 氯马昔巴特 相关的临床试验NCT07389031
Maralixibat for the trEatment of inTrAhePatic cHOlestasis of pRegnancy (METAPHOR)
NCT07293897
A Cohort Study to Investigate the Risk of Liver Disorder of Livmarli Oral Solution in Patients With Japan Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) Registered in the Comprehensive and Informative Registry System for Childhood Liver Disease (CIRCLe)
NCT07290257
Long-Term Low-Intervention SafEty and Clinical Outcomes Clinical Study of LivmArli® in Patients With Alagille Syndrome or Progressive Familial Intrahepatic Cholestasis in the European Union (LEAP-EU)
100 项与 氯马昔巴特 相关的临床结果
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100 项与 氯马昔巴特 相关的转化医学
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100 项与 氯马昔巴特 相关的专利（医药）
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52
项与 氯马昔巴特 相关的文献（医药）2026-07-01ALIMENTARY PHARMACOLOGY & THERAPEUTICS
Review Article: Ileal Bile Acid Transport (
IBAT
) Inhibitors as an Emerging Treatment for Cholestatic Liver Disease
Review
作者: Joshi, Deepak ; Peverelle, Matthew ; Shah, Sital ; Peverelle, James ; Greener, Kate ; Nayagam, Jeremy S. ; Campbell, Samantha M. A.
ABSTRACT:
Background:
Cholestatic liver diseases such as Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) cause significant morbidity and mortality. Chronic pruritus is common, debilitating, and impairs health‐related quality of life. Recently, pharmacological inhibition of the ileal bile acid transporter (IBAT) has emerged as a therapeutic target.
Aims:
To review the current landscape of IBAT inhibitors, summarise emerging clinical data, discuss their role in the treatment of cholestatic liver diseases and future directions for their development and use.
Methods:
This narrative review summarises current data on IBAT inhibitors, exploring their mechanisms, efficacy, and safety across ALGS, PFIC, PBC and PSC. References were identified through searches of PubMed from January 2000 to August 2025.
Results:
In phase 2 and 3 trials involving paediatric patients with ALGS and PFIC, IBAT inhibitors (odevixibat and maralixibat) significantly reduced pruritus and serum bile acid concentrations. In post hoc analysis, responders demonstrated improved event‐free and transplant‐free survival compared to historic control cohorts. The phase 3 trial of linerixibat in PBC demonstrated an improvement in pruritus compared to placebo. Mild to moderate gastrointestinal side effects, most commonly diarrhoea and abdominal discomfort, are common with IBAT inhibition, particularly in PBC and PSC.
Conclusions:
IBAT inhibitors represent the first upstream pharmacotherapy targeting enterohepatic bile acid recirculation and are effective at reducing pruritus in ALGS and PFIC. Their role in PBC and PSC is promising yet undefined. Long‐term studies are needed to assess effects on fibrosis progression, hepatocellular carcinoma risk and transplant‐free survival.
2026-07-01JOURNAL OF PEDIATRICS
Maralixibat Improves Xanthomas and Hypercholesterolemia in Children with Alagille Syndrome: A Post Hoc Integrated Analysis From Two Clinical Trials
Article
作者: Chen, Cece ; Karnsakul, Wikrom ; Hoskins, Brett J ; Mogul, Douglas B
OBJECTIVE:
To characterize baseline xanthoma prevalence and severity in Alagille syndrome, assess their impact on quality of life, evaluate longitudinal changes in xanthoma burden with maralixibat therapy, and identify clinical and biochemical predictors of treatment response in children enrolled across 2 clinical trials.
STUDY DESIGN:
This integrated analysis from the ICONIC and ITCH trials assessed xanthoma severity using the Clinician Xanthoma Scale (CXS). Response was defined as a ≥1-point reduction from baseline in CXS. Outcomes included changes in serum lipids, serum bile acids (sBA), pruritus, and Pediatric Quality of Life Inventory scores through week 96. Baseline characteristics were compared across xanthoma severity groups using the Jonckheere-Terpstra test for continuous variables and the Cochran-Armitage trend test for categorical variables. The changes in xanthoma severity category (3 levels) from baseline to weeks 48 and 96 were tested using Bowker's test of symmetry. Change from baseline (CFB) of continuous outcomes was tested using the Wilcoxon signed-rank test. Differences in CFB of outcomes between xanthoma responders and nonresponders were evaluated using the Wilcoxon rank-sum test for continuous variables and Fisher exact test for categorical outcomes.
RESULTS:
Among 63 children (ICONIC, n = 29; ITCH, n = 34), 43% had xanthomas at baseline. Higher CXS was associated with younger age (P = .03); elevated sBA (P < .001), bilirubin (P < .001), alanine aminotransferase (P = .008), and gamma-glutamyl transferase (P = .02); and worse lipid profiles (total cholesterol, P < .001; high-density lipoprotein, P < .001). Quality of life declined with increasing CXS. After 96 weeks of follow-up (ICONIC, n = 18; ITCH, n = 17), mean CXS decreased (P = .004), and CXS 0 prevalence increased from 60% to 86% (P = .03). There was a significant decline in cholesterol (mean CFB, -55 mg/dL, P < .001) and sBA (mean CFB, -85 μmol/L, P < .001). Among participants with baseline CXS ≥1 (n = 14; ICONIC, n = 8; ITCH, n = 6), 71% and 64% were xanthoma responders at weeks 48 and 96. Responders had greater cholesterol reductions at week 48 (mean CFB, -189 vs -11 mg/dL, P = .005) and more frequent pruritus improvement (94% vs 68%, P = .047).
CONCLUSIONS:
Maralixibat may reduce xanthoma burden and improve metabolic parameters, supporting therapeutic potential of the drug for xanthomas in Alagille syndrome.
CLINICAL TRIAL REGISTRATION:
This is not a clinical trial, but an integrated analysis of 2 trials; these trials are registered with ClinicalTrials.gov. • ICONIC: NCT02160782, https://clinicaltrials.gov/study/NCT02160782 • ITCH: NCT02057692, https://clinicaltrials.gov/study/NCT02057692 • IMAGINE-II (long-term extension of ITCH): NCT02117713, https://clinicaltrials.gov/study/NCT02117713.
2026-03-01Liver International
Ileal Bile Acid Transporter Inhibitors in Cholestasis: Potential for More Than Just Paediatrics?
Review
作者: Boyer, James Lorenzen ; Krishna, Mahesh
ABSTRACT:
Ileal bile acid transporter inhibitors (IBATi) are a new, attractive therapeutic mechanism to alter the enterohepatic circulation through depletion of the bile acid pool by blocking bile acid reuptake in the ileum leading to improvements in pruritus and liver function in cholestatic liver diseases. These drugs may also have an impact on immunity, the gut microbiome, and motility. IBATi are approved in Japan for the treatment of idiopathic chronic constipation. There are two IBATi, maralixibat and odevixibat, that have been extensively investigated in clinical trials and are FDA approved for cholestatic pruritus in progressive familial intrahepatic cholestasis and Alagille syndrome. Clinical trials exploring IBATi in other cholestatic conditions, such as biliary atresia, primary biliary cholangitis, and primary sclerosing cholangitis, are currently ongoing. In this review, we will outline the emerging data regarding the physiology and mechanism of action for the IBATi class, an overview of clinical trials that led to the approval of maralixibat and odevixibat, ongoing clinical trials in adult cholestatic liver diseases, and the future of this drug class in systemic apical sodium bile acid transporter inhibitors.
100 项与 氯马昔巴特 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 |
|---|---|---|---|
| 进行性家族性肝内胆汁淤积症 | 欧盟 | 2024-07-08 | |
| 进行性家族性肝内胆汁淤积症 | 冰岛 | 2024-07-08 | |
| 进行性家族性肝内胆汁淤积症 | 列支敦士登 | 2024-07-08 | |
| 进行性家族性肝内胆汁淤积症 | 挪威 | 2024-07-08 | |
| 阿拉吉欧综合症 | 欧盟 | 2022-12-09 | |
| 阿拉吉欧综合症 | 冰岛 | 2022-12-09 | |
| 阿拉吉欧综合症 | 列支敦士登 | 2022-12-09 | |
| 阿拉吉欧综合症 | 挪威 | 2022-12-09 | |
| 胆汁淤积性瘙痒 | 美国 | 2021-09-29 |
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 |
|---|---|---|---|---|
| 胆汁淤积性肝病 | 临床3期 | 美国 | 2024-10-14 | |
| 胆汁淤积性肝病 | 临床3期 | 巴西 | 2024-10-14 | |
| 胆汁淤积性肝病 | 临床3期 | 加拿大 | 2024-10-14 | |
| 胆汁淤积性肝病 | 临床3期 | 法国 | 2024-10-14 | |
| 胆汁淤积性肝病 | 临床3期 | 德国 | 2024-10-14 | |
| 胆汁淤积性肝病 | 临床3期 | 意大利 | 2024-10-14 | |
| 胆汁淤积性肝病 | 临床3期 | 黎巴嫩 | 2024-10-14 | |
| 胆汁淤积性肝病 | 临床3期 | 波兰 | 2024-10-14 | |
| 胆汁淤积性肝病 | 临床3期 | 西班牙 | 2024-10-14 | |
| 胆汁淤积性肝病 | 临床3期 | 英国 | 2024-10-14 |
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临床结果
临床结果
适应症
分期
评价
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临床2期 | 63 | 蓋鏇鹹願夢壓蓋襯淵鏇(壓鑰獵齋壓簾觸範鏇網) = 鬱蓋網遞糧蓋鹹鏇襯齋 夢構網餘餘壓鑰簾鏇網 (壓膚襯廠衊顧廠窪齋觸 ) 更多 | 积极 | 2026-07-01 | |||
临床2期 | 27 | (Maralixibat: ALGS) | 簾簾選蓋鹹構艱繭壓鹽 = 願膚窪餘淵艱憲範顧廠 積蓋簾選壓觸鬱鏇範襯 (壓範膚製窪繭鹹鏇鹽壓, 鹽糧鬱願簾構範願選蓋 ~ 齋憲鹽餘襯淵選醖衊築) 更多 | - | 2026-06-02 | ||
PFIC+Maralixibat (Maralixibat: PFIC) | 簾簾選蓋鹹構艱繭壓鹽 = 醖積範觸獵艱築夢淵鬱 積蓋簾選壓觸鬱鏇範襯 (壓範膚製窪繭鹹鏇鹽壓, 簾構憲蓋願襯艱觸築憲 ~ 繭鹹遞憲構築鹽齋積鏇) 更多 | ||||||
临床2期 | 52 | (Maralixibat: ALGS) | 願齋糧範簾選鏇夢鹽衊 = 襯襯夢鹹糧觸鏇獵鏇窪 構壓鑰鹽糧壓憲齋膚廠 (衊蓋顧積積蓋淵遞築繭, 願醖鹹醖觸鏇齋簾願繭 ~ 餘窪夢蓋獵鹽鑰淵選蓋) 更多 | - | 2025-12-08 | ||
PFIC+Maralixibat (Maralixibat: PFIC) | 願齋糧範簾選鏇夢鹽衊 = 築淵醖顧糧積鏇遞淵願 構壓鑰鹽糧壓憲齋膚廠 (衊蓋顧積積蓋淵遞築繭, 觸蓋簾選壓淵繭構衊憲 ~ 顧齋遞蓋衊選獵醖襯簾) 更多 | ||||||
临床3期 | 64 | 憲願願鏇齋憲壓窪簾觸(衊製淵鏇網繭製夢衊淵) = 鬱夢餘衊遞糧艱膚淵製 壓鹽廠觸鏇齋蓋壓製觸 (膚衊鹹積願淵齋襯艱網, -2.2 ~ -1.4) | 积极 | 2025-04-10 | |||
Placebo | 憲願願鏇齋憲壓窪簾觸(衊製淵鏇網繭製夢衊淵) = 艱窪築簾窪衊鹹衊齋糧 壓鹽廠觸鏇齋蓋壓製觸 (膚衊鹹積願淵齋襯艱網, -1.0 ~ -0.2) | ||||||
临床2期 | 29 | 醖鬱衊築衊構製餘夢願(製憲鹽構壓艱窪製壓構) = 餘願艱夢窪範簾繭構繭 廠糧衊積範獵壓艱鏇鏇 (鑰窪網膚獵鹹鬱鹽繭憲, -0.3 ~ 0.7) | 积极 | 2025-04-10 | |||
Placebo | 醖鬱衊築衊構製餘夢願(製憲鹽構壓艱窪製壓構) = 鹽繭範糧範築積廠襯憲 廠糧衊積範獵壓艱鏇鏇 (鑰窪網膚獵鹹鬱鹽繭憲, 1.2 ~ 2.1) | ||||||
临床2期 | 75 | (Double Blind - Maralixibat) | 齋積餘觸製蓋淵觸網鬱(顧鏇獵壓鹹鬱衊糧夢膚) = 顧構範鑰餘鹹壓夢蓋艱 衊網構鬱遞蓋蓋願積壓 (餘餘選壓襯鑰築衊艱構, 0.853) 更多 | - | 2025-02-17 | ||
Placebo (Double Blind - Placebo) | 齋積餘觸製蓋淵觸網鬱(顧鏇獵壓鹹鬱衊糧夢膚) = 鹹選淵餘積窪網獵獵糧 衊網構鬱遞蓋蓋願積壓 (餘餘選壓襯鑰築衊艱構, 0.947) 更多 | ||||||
临床3期 | 7 | 築顧繭衊夢壓網壓構艱(製鏇壓醖夢襯廠壓壓鏇) = 網觸糧鏇糧窪膚衊夢糧 鏇遞膚築積憲衊簾築壓 (糧糧選鏇夢構蓋齋衊網, 艱鹹襯鬱網獵憲鬱憲壓 ~ 構鏇鹽範蓋網遞觸繭鏇) 更多 | - | 2024-10-15 | |||
临床3期 | 5 | (Primary Cohort: TAK-625) | 糧壓積遞衊襯糧醖鹽糧(遞鬱築衊願鹽蓋顧鬱鹹) = 壓觸繭選簾糧糧鹽鑰衊 膚襯淵製鏇築獵鏇齋蓋 (顧簾窪願築築窪壓衊憲, 醖醖壓夢壓襯觸積夢築 ~ 鑰憲艱鹹齋繭餘齋願網) 更多 | - | 2024-09-24 | ||
(Supplemental Cohort: TAK-625) | 糧壓積遞衊襯糧醖鹽糧(遞鬱築衊願鹽蓋顧鬱鹹) = 簾壓繭鹹廠鏇選網鹹觸 膚襯淵製鏇築獵鏇齋蓋 (顧簾窪願築築窪壓衊憲, 製齋廠築齋艱範窪繭選 ~ 簾憲獵遞壓簾獵鏇廠艱) 更多 | ||||||
临床2期 | 76 | 鑰選鑰淵鏇淵繭觸淵淵(願網觸簾獵齋廠顧製鑰) = 鹽範構糧壓淵獵醖憲獵 繭鹹鬱淵顧繭觸顧鑰鬱 (獵壓壓鏇顧淵簾襯選憲 ) 更多 | 积极 | 2024-06-07 | |||
N/A | - | 鏇憲繭憲網鏇膚憲壓簾(艱遞鹹獵積憲範蓋鑰憲) = in pruritus severity, serum bile acid (sBA) levels, total bilirubin and growth following up to two years of LIVMARLI treatment. Similar improvements in pruritus and sBA were seen in patients originally randomized to placebo who received LIVMARLI in the open-label study. 襯鑰蓋壓糧選壓壓繭構 (夢選夢窪範範繭憲餘願 ) | 积极 | 2024-05-18 |
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