Randomized Double-Blind Placebo-Controlled Phase 3 Study to Evaluate the Efficacy and Safety of Maralixibat in the Treatment of Participants With Cholestatic Pruritus

The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric and adult participants who have cholestatic liver disease with pruritus that has been refractory to other therapies, and who have no other treatment options.

开始日期2024-10-14

申办/合作机构

Mirum Pharmaceuticals, Inc.

NCT06413368

/ Not yet recruiting临床2/3期IIT

Maralixibat in Patients With Cystic Fibrosis and Constipation, A Within-Subjects Pilot Study

Chronic constipation is a feature of children with cystic fibrosis (CF). This is postulated to be a result of inhibition of secretory activity of the gastrointestinal luminal cells due to ineffective chloride channel function. Typical laxatives that work as osmotic agents fail to produce adequate relief in this population.
Maralixibat is a non-systemic bile acid transport inhibitor (IBATi) that acts by interrupting bile acid reabsorption in the ileum thus interrupting the normal enterohepatic circulation. This interruption results in a larger volume of bile acids reaching the colon and being excreted in stool. Bile acids are known to decrease bowel transit time, increase mucosal permeability and secretions, as well as alter gut microbiota resulting in diarrhea.
The overarching hypothesis of the study is that Maralixibat will improve stool consistency in children (Age <18 years) with cystic fibrosis and constipation (Bristol Stool Scale <4). Specifically, we aim to test the hypothesis that IBATi improves the consistency of stool to Bristol scale >4 in children with CF and constipation.
We will recruit a total of 20 patients with CF and constipation (defined as Bristol Stool Scale <4 for 1 week prior to enrollment while on a stable laxative regimen for at least 4 weeks.) Design is a 'Within-Subjects' study by which each enrolled patient will take Maralixibat for 2 weeks total in addition to their stable laxative regimen during the study. Stool consistency & ease of defecation will be recorded before and during the study period by families of enrolled patients via materials provided by the investigators. Stool consistency and ease of defecation will be compared before and after initiation of Maralixibat.
The primary endpoint: Improvement in stool consistency to Bristol scale >4 in children with CF and constipation. The secondary endpoint: Improvement in ease of defecation in children with CF and constipation. This will be measured via survey using a standardized scale (Bristol Stool Scale) and questionnaires developed by the research team. Analysis will involve comparison of pre-intervention to post-intervention stool consistency & survey

开始日期2024-07-01

申办/合作机构

Children's Hospital Los Angeles

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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项与氯马昔巴特相关的文献（医药）

2025-11-01·JOURNAL OF LIPID RESEARCH

Carrier cross-reactivities of the bile acid reabsorption inhibitors elobixibat, linerixibat, maralixibat, and odevixibat

Article

作者: Neubauer, Anita ; Geyer, Joachim ; Fühler, Bärbel ; Billo, Veronica ; Neelen, Christopher ; Wannowius, Marie ; Hagos, Yohannes

The bile acid reabsorption inhibitors (BARIs) elobixibat, maralixibat, and odevixibat are clinically used inhibitors of the intestinal bile acid transporter ASBT (SLC10A2). An additional BARI compound, linerixibat, is still under clinical development. In the present study, potential cross-reactivities against the closely related hepatic bile acid carrier and hepatitis B virus entry receptor NTCP (SLC10A1), as well as the steroid sulfate uptake carrier SOAT (SLC10A6) were analyzed. All BARIs potently inhibited ASBT (IC₅₀ = 0.1-1.0 μM). Among them, elobixibat, maralixibat, and odevixibat also inhibited SOAT (IC₅₀ = 3.2-5.9 μM) and NTCP (IC₅₀ = 10-99 μM). Furthermore, all four BARIs inhibited the hepatic drug transporters OATP1B1, OATP1B3, and OATP2B1 (IC₅₀ = 1.6-29 μM). Notably, ASBT inhibition by linerixibat was reversible upon washout, while maralixibat and odevixibat induced full and sustained ASBT inhibition even after removal of the inhibitor and inhibitor-free incubation over 240 min. Elobixibat and the pan-SLC10 inhibitor troglitazone revealed an intermediate effect. The ASBT S294T/I295V double mutation increased the inhibitory potency of linerixibat, suggesting a role of this domain for linerixibat binding. In contrast, this mutation had no significant effect on the ASBT inhibition by elobixibat, maralixibat, and odevixibat, indicating distinct binding sites. In conclusion, the analyzed BARIs revealed carrier cross-reactivities with NTCP, SOAT, and members of the OATP family, but behaved differently regarding their time-dependent inhibition and potential inhibitor binding sites.

2025-11-01·CLINICAL THERAPEUTICS

Indirect Comparison of Maralixibat and Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis

Article

作者: Darlington, Oliver ; Gosden, Toby ; Evers, Elise ; Quadrado, Lucia ; Howard, Robin ; Lacey, Guy

PURPOSE:

Recent clinical trials provide evidence of the efficacy and safety of 2 ileal bile acid transporter inhibitors, maralixibat and odevixibat, for the treatment of children with progressive familial intrahepatic cholestasis (PFIC). However, no head-to-head trial currently exists assessing the efficacy of these 2 treatments. The objective of this study was to generate estimates of comparative efficacy and safety between maralixibat and odevixibat to inform optimal practices for the treatment of children with PFIC.

METHODS:

Two phase 3 randomized placebo-controlled trials, MARCH-PFIC (maralixibat: n = 16; placebo: n = 19) and PEDFIC-1 (odevixibat: n = 42; placebo: n = 20), were included in an indirect treatment comparison (ITC) anchored by the placebo arms of each trial. Using patient-level data from MARCH-PFIC and published aggregate data from PEDFIC-1, we generated estimates of comparative efficacy for endpoints that were consistent between trials, including the proportion of patients achieving a serum bile acid (sBA) response and change from baseline in sBA concentration.

FINDINGS:

Comparisons of the proportion of sBA responders indicated that maralixibat was significantly more efficacious than odevixibat (120 µg/kg), with an estimated treatment difference of 32.3% (95% CI, 1.1% to 63.4%, P = 0.043). In addition, point estimates for change from baseline in sBA concentration and total bilirubin trended in favor of maralixibat. These findings were robust to adjustments for imbalances in patient demographic characteristics between trials. No statistically significant differences were observed for alanine transaminase, aspartate transaminase, or gamma-glutamyl transferase. The safety profiles of maralixibat and odevixibat were comparable, although adverse events associated with maralixibat were typically milder than those with odevixibat (mild: 75% vs 45%; moderate: 25% vs 31%; severe: 0% vs 7%).

IMPLICATIONS:

These findings suggest that maralixibat provides additional clinical benefit for children with PFIC compared with odevixibat in terms of increasing the proportion of sBA responders. Further studies are needed to compare the ability of maralixibat and odevixibat to reduce pruritus and improve long-term outcomes, including patients' quality of life.

2025-09-01·JOURNAL OF HEPATOLOGY

Use of maralixibat in severe and recurrent intrahepatic cholestasis of pregnancy with concurrent benign recurrent intrahepatic cholestasis

Letter

作者: Katzenstein, Cecilia ; Kushner, Tatyana ; O'Kula, Susanna ; Rodriguez, Nina

265

项与氯马昔巴特相关的新闻（医药）

2025-11-07

·ir.mirumpharma.com

Mirum Pharmaceuticals Showcases Leadership in Liver Disease Innovation With Data From LIVMARLI® and Volixibat Clinical Programs at AASLD’s The Liver Meeting®

New data reinforce Mirum’s commitment to advancing therapies and improving the lives of patients across diverse rare liver diseases. FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that multiple presentations featuring data from its LIVMARLI (maralixibat) and volixibat clinical programs will be shared during the American Association for the Study of Liver Diseases’ (AASLD) The Liver Meeting®, taking place November 7-11, 2025, in Washington, D.C. The presentations highlight Mirum’s continued progress in expanding the scientific and clinical foundation for new approaches to treating adult and pediatric rare liver diseases, including new findings from the VANTAGE trial and real-world studies. Oral Presentation A key highlight at this year’s meeting will be an oral presentation featuring additional analyses from the VANTAGE trial evaluating volixibat in adults with primary biliary cholangitis (PBC). Abstract #0215: Volixibat Leads to Improvements in Fatigue and Sleep for Adults with Primary Biliary Cholangitis: Data from VANTAGE Trial Monday, November 10, 2025, 3:00-3:15 pm during the ‘Novel Therapies and Monitoring in Cholestatic Liver Disease’ session, Room 145, Level 1, Convention Center Presented by Kris Kowdley, Liver Institute Northwest, Seattle, Washington, USA Poster Presentations In addition to new volixibat data, several abstracts highlight real-world experience with maralixibat and insights from genetic testing in cholestasis. Abstract #4411: Maralixibat, an Ileal Bile Acid Transporter Inhibitor, Reduces Cholestatic Pruritus in PSC: Real-World Experience Monday, November 10, 2025, 1:00-2:00 pm Posters and Exhibit Hall, Level 2, Convention Center Presented by Dr. Adrielly Martins, University of Miami, Florida, USA Abstract #4412: Clinical Utility of Comprehensive Genetic Testing in Adult Cholestasis: Insights from 856 Panel Tests Monday, November 10, 2025, 1:00-2:00 pm Posters and Exhibit Hall, Level 2, Convention Center Presented by Dr. Tiziano Pramparo, Mirum Pharmaceuticals, Inc., Foster City, California, USA Abstract #4415: Exploring the Role of Ileal Bile Acid Transporter Inhibitors, Like Volixibat, in the Regulation of IL-31 and Serum Bile Acids in Patients with Primary Biliary Cholangitis: Preliminary Results from the VANTAGE Trial Monday, November 10, 2025, 1:00-2:00 pm Posters and Exhibit Hall, Level 2, Convention Center Presented by Dr. Alan Bonder, Beth Israel Deaconess Medical Center, Boston, Massachusetts, USA Abstract #4511: Physician Reported Assessment of Pruritus Amongst Patients with PSC: Results from a Real-World Survey in France, Germany, Italy and the United States Monday, November 10, 2025, 1:00-2:00 pm Posters and Exhibit Hall, Level 2, Convention Center Presented by Dr. Marlyn Mayo, UT Southwestern Medical Center, Dallas, Texas, USA Abstract #4532: Impact of Maralixibat on Caregiver Burden for Patients with Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis: Baseline and 6-month Follow-up Monday, November 10, 2025, 1:00-2:00 pm Posters and Exhibit Hall, Level 2, Convention Center Presented by Dr. Natasha Dilwali, Columbia University Irving Medical Center, New York, New York, USA Fireside Discussion Mirum will also host an educational fireside discussion addressing current approaches to Progressive Familial Intrahepatic Cholestasis (PFIC) diagnosis and management. PFIC Diagnosis and Treatment Fireside Discussion (Mirum-Sponsored) During The Liver Meeting, Mirum will host a session featuring Dr. Robert Gish, Loma Linda University, focused on the recognition and diagnosis of PFIC, including the role of genetic testing and disease management considerations for adults. The discussion will also include a review of clinical data for LIVMARLI in PFIC. Sunday, November 9, 2025, 1:30-2:00 pm Exhibit Hall TLM Theater Mirum’s presence at The Liver Meeting underscores the company’s commitment to advancing therapies that improve patient outcomes across rare liver diseases. Full abstracts are available on the AASLD website. Following the conference, detailed analyses will be posted in the Publications and Presentations section of Mirum’s website. About LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets LIVMARLI® (maralixibat) is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI.com. LIVMARLI has received orphan designation for ALGS and PFIC. LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website. IMPORTANT SAFETY INFORMATION Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein. LIVMARLI can cause side effects, including Liver injury. Changes in certain liver tests are common in patients with ALGS and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal. Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you. A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat is common in patients with Alagille syndrome and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects. US Prescribing Information EU SmPC Canadian Product Monograph About Volixibat Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study), and primary biliary cholangitis (PBC) (VANTAGE study). In 2024, Mirum announced positive interim results from the Phase 2b VANTAGE study showing statistically significant improvement in pruritus as well as meaningful reductions in serum bile acids and improvements in fatigue for patients treated with volixibat. No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate. Volixibat has been granted breakthrough therapy designation for the treatment of PBC. About Mirum Pharmaceuticals, Inc. Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution/LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI® (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum is conducting the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum has also initiated a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X). Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the Company’s planned participation at a scientific congress, Mirum’s leadership in liver disease and some anticipated scientific presentations of data from Mirum’s LIVMARLI and volixibat studies at the American Association for the Study of Liver Diseases. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “expected,” “will,” “could,” “would,” “guidance,” “potential,” “continue” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum’s business in general, the impact of geopolitical and macroeconomic events, and the other risks described in Mirum’s Annual Report for the year ended December 31, 2024, filed with the Securities and Exchange Commission on February 26, 2025, and subsequent filings with the Securities and Exchange Commission, which are available at www.sec.gov. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. Source: Mirum Pharmaceuticals, Inc. Investor Contact: Andrew McKibben ir@mirumpharma.com Media Contact: Meredith Kiernan media@mirumpharma.com

临床2期临床结果上市批准突破性疗法孤儿药

2025-11-05

·北海康成

北海康成三款罕见病产品均纳入2026年度北京普惠健康保

北海康成制药有限公司（以下简称“北海康成”，股票代码“1228.HK”），是一家在中国领先并专注罕见疾病领域的全球化的生物制药公司，致力于创新疗法的研究、开发和商业化。 2026年度“北京普惠健康保”保障详情已于近期公布，北海康成三款罕见病产品均已纳入，其中戈芮宁®首次亮相“惠民保”。北京将罕见病药物纳入惠民保的举措，不仅是对本地患者的精准支持，更以头部城市的实践为标杆，或将带动更多代表性城市在惠民保的罕见病保障维度上加快布局。此次三款产品均纳入《特药2目录（37种）》。戈芮宁于今年5月13日获批上市，是国内首个本土自主研发适用于12岁及以上青少年和成人I型和Ⅲ型戈谢病患者的长期酶替代疗法，也是我国首个通过生物制品分段生产检查的创新生物药。7月2日，戈芮宁在上海交通大学医学院附属新华医院开出首方，标志着该药物正式进入临床应用，未来将大大提升国内患者用药可及。另外两款产品分别是治疗3个月及以上阿拉杰里（ALGS）患者胆汁淤积性瘙痒的迈芮倍®和治疗黏多糖贮积症II型（MPS II）的海芮思®。海芮思和迈芮倍分别是国内在MPS II和ALGS获批的首个针对性治疗。迈芮倍于2023年获批，2024开始商业化并于同年首次纳入惠民保，截止目前已纳入了31个城市惠民保，1个商业健康险。海芮思从2020年首次纳入上海沪惠保，截止目前纳入了85个城市惠民保，1个商业健康险。《特药2目录（37种）》特药2责任：①保障责任为经指定医疗机构专科医生诊断并开具处方，在指定医疗机构普通门诊药房或院外药店购买药品。②医院范围为中华人民共和国境内（不包括香港、澳门、台湾地区）的医保定点医疗机构及药店。③特药2保额为50万元/年。④免赔额为0元。⑤给付比例为健康人群65%，特定既往症人群35%。这三款罕见病药物纳入2026年度北京普惠健康保，将为北京的罕见病患者带来了新的用药选择和希望。注：点击文末"阅读原文"查看 2026年度“北京普惠健康保”保障详情向上滑动阅览北海康成制药有限公司（以下简称“北海康成”，股票代码1228.HK）是一家在中国领先并专注罕见疾病领域的全球化的生物制药公司，致力于创新疗法的研究、开发和商业化。公司目前拥有10个具有可观市场潜力的药物资产组合，其中包括3种已获批上市产品和7个在研药物。这些产品均针对一些较普遍的罕见疾病，比如亨特综合征（黏多糖贮积症II型）和其他溶酶体贮积症、补体介导的疾病、血友病 A、代谢紊乱、罕见的胆汁淤积性肝病、神经肌肉疾病。北海康成的新一代基因技术研发中心实验室正在开发针对罕见遗传病，包括庞贝氏症、法布雷病、杜氏肌肉营养不良症（DMD）和其他神经肌肉疾病的新型及潜在治愈性的基因疗法，并与世界领先的研究人员和生物技术公司合作。公司的全球合作伙伴包括不限于 Apogenix、GC Pharma、Mirum、药明生物、Privus、华盛顿大学医学部和Scriptr Global 等。如需了解更多关于北海康成制药有限公司的信息，请访问：http://www.canbridgepharma.com/ 前瞻性声明：本文中作出的前瞻性陈述仅与本文作出陈述之日的事件或信息有关。除非法律要求，否则我们不承担公开更新或修改任何前瞻性陈述的义务，无论是由于新信息、未来事件或其他原因，在做出陈述的数据之后或反映意外事件的发生情况。您应完整阅读本文，并了解我们未来的实际结果或表现可能与我们的预期存在重大差异。在本文中，我们或我们的任何董事或我们公司的意图的陈述或引用均在本文发表之日作出。这些意图中的任何一个都可能随着未来的发展而改变。联系我们：投资者关系： ir@canbridgepharma.com

上市批准申请上市

2025-11-03

·Business Wire

Mirum Pharmaceuticals to Participate in Upcoming Investor Conferences

FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical company focused on the identification, acquisition, development and commercialization of novel therapies for debilitating rare and orphan diseases, today announced that it will participate in the following upcoming investor conferences: 2025 Stifel Healthcare Conference Company presentation - Tuesday, November 11 th , starting at 8:40 a.m. ET Company presentation - Tuesday, November 11 th , starting at 8:40 a.m. ET J.P. Morgan U.S. Opportunities Forum 2025 on Wednesday, November 12 th Visit the Investors and Media section of Mirum’s corporate website for webcast links and additional information. About Mirum Pharmaceuticals, Inc. Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution/LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum is conducting the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2b VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum has also initiated a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and X.

临床2期上市批准突破性疗法临床3期

100 项与氯马昔巴特相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
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研发状态

批准上市

10 条最早获批的记录,

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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	Mirum Pharmaceuticals, Inc.	2024-07-08
进行性家族性肝内胆汁淤积症	冰岛	Mirum Pharmaceuticals, Inc.	2024-07-08
进行性家族性肝内胆汁淤积症	列支敦士登	Mirum Pharmaceuticals, Inc.	2024-07-08
进行性家族性肝内胆汁淤积症	挪威	Mirum Pharmaceuticals, Inc.	2024-07-08
阿拉吉欧综合症	欧盟	Mirum Pharmaceuticals, Inc.	2022-12-09
阿拉吉欧综合症	冰岛	Mirum Pharmaceuticals, Inc.	2022-12-09
阿拉吉欧综合症	列支敦士登	Mirum Pharmaceuticals, Inc.	2022-12-09
阿拉吉欧综合症	挪威	Mirum Pharmaceuticals, Inc.	2022-12-09
胆汁淤积性瘙痒	美国	Mirum Pharmaceuticals, Inc.	2021-09-29

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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	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	巴西	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	加拿大	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	法国	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	德国	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	意大利	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	黎巴嫩	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	墨西哥	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	波兰	Mirum Pharmaceuticals, Inc.	2024-10-14
胆汁淤积性肝病	临床3期	沙特阿拉伯	Mirum Pharmaceuticals, Inc.	2024-10-14

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT02160782 (FDA_CDER) 人工标引	临床2期	阿拉吉欧综合症 \| 瘙痒	29	Maralixibat	築淵憲鬱廠簾齋齋構獵(鏇選鬱繭廠顧鏇鹹窪鬱) = 窪夢遞鹽鑰憲簾製膚築廠製襯構鏇鏇構製鹹艱 (襯醖憲襯壓鑰蓋艱衊壓, -0.3 ~ 0.7)	积极	2025-04-10
				Placebo	築淵憲鬱廠簾齋齋構獵(鏇選鬱繭廠顧鏇鹹窪鬱) = 鹹網衊範獵廠糧窪鏇夢廠製襯構鏇鏇構製鹹艱 (襯醖憲襯壓鑰蓋艱衊壓, 1.2 ~ 2.1)
NCT03905330 (FDA_CDER) 人工标引	临床3期	进行性家族性肝内胆汁淤积症	64	Maralixibat	願選鑰壓簾窪窪繭蓋製(鹹觸艱衊繭醖簾夢選壓) = 積醖鑰餘獵積齋網遞鑰襯鑰築夢壓艱製廠憲膚 (願構網觸鏇鑰夢鏇鬱製, -2.2 ~ -1.4)	积极	2025-04-10
				Placebo	願選鑰壓簾窪窪繭蓋製(鹹觸艱衊繭醖簾夢選壓) = 製壓鹽觸選襯獵構繭構襯鑰築夢壓艱製廠憲膚 (願構網觸鏇鑰夢鏇鬱製, -1.0 ~ -0.2)
NCT04524390 (EMBARK, CTgov)	临床2期	胆道闭锁	75	Maralixibat (Double Blind - Maralixibat)	窪製遞構醖憲獵獵築齋(鹹糧壓製膚壓廠壓鹽鹹) = 襯遞願築蓋觸獵廠獵獵齋壓簾網構繭顧衊鹽觸 (觸鏇願築憲願構糧築壓, 0.853) 更多	-	2025-02-17
				Placebo (Double Blind - Placebo)	窪製遞構醖憲獵獵築齋(鹹糧壓製膚壓廠壓鹽鹹) = 網齋製襯蓋鏇遞襯醖遞齋壓簾網構繭顧衊鹽觸 (觸鏇願築憲願構糧築壓, 0.947) 更多
NCT05543174 (CTgov)	临床3期	阿拉吉欧综合症	7	TAK-625	襯齋夢願遞艱構餘壓衊(構構鏇廠餘鏇膚窪襯簾) = 觸構鏇醖襯鏇範觸齋憲糧鏇鏇襯獵鑰網鹹觸鑰 (鹽獵製醖積積壓廠築選, 鹹簾窪憲積醖壓襯鹽簾 ~ 膚積鏇淵蓋餘餘遞遞廠) 更多	-	2024-10-15
NCT05543187 (CTgov)	临床3期	进行性家族性肝内胆汁淤积症	5	TAK-625 (Primary Cohort: TAK-625)	願鏇夢鬱廠鏇壓願廠鏇(觸襯積蓋鬱膚艱築鹽壓) = 膚夢衊鑰獵壓襯選糧醖顧網壓鹽構選憲觸範積 (選窪獵艱夢鹹網鹹鏇淵, 餘淵蓋膚醖夢淵廠網憲 ~ 壓壓廠築壓簾窪鬱遞艱) 更多	-	2024-09-24
				TAK-625 (Supplemental Cohort: TAK-625)	願鏇夢鬱廠鏇壓願廠鏇(觸襯積蓋鬱膚艱築鹽壓) = 窪廠獵淵構選選範鑰鑰顧網壓鹽構選憲觸範積 (選窪獵艱夢鹹網鹹鏇淵, 醖醖築衊範築蓋淵築艱 ~ 鹽繭顧獵壓遞範鹹鬱築) 更多
NCT04168385 (MERGE, NEWS)	临床2期	阿拉吉欧综合症	76	马昔巴特	積齋襯構範衊鹹選網衊(鏇繭淵壓遞選廠構鹽構) = 襯網蓋夢範選顧糧襯夢壓鏇選鏇廠願願選窪鬱 (襯壓襯觸壓醖獵鏇窪齋 ) 更多	积极	2024-06-07
MARCH-ON (Biospace) 人工标引	N/A	进行性家族性肝内胆汁淤积症	-	LIVMARLI	遞鹽蓋齋餘醖願壓鑰醖(繭鹹餘蓋蓋膚遞獵遞觸) = 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. 構鑰構觸鏇蓋齋獵鹽獵 (蓋鏇範壓製鹽齋廠繭獵 )	积极	2024-05-18
NCT03905330 (MARCH-PFIC, Pubmed) 人工标引	临床3期	进行性家族性肝内胆汁淤积症 biallelic, non-truncated BSEP deficiency \| biallelic FIC1 \| MDR3 ... 更多	93	Maralixibat	積壓膚窪醖壓廠觸構艱(鑰獵齋鹽艱艱淵壓艱淵) = 憲糧願觸簾製築壓淵齋鏇壓鹽製憲獵製艱膚顧 (夢製廠憲艱顧窪築鑰壓 ) 更多	积极	2024-05-06
				Placebo	鑰糧積廠衊壓壓構襯遞(鏇鏇餘獵繭遞窪夢製願) = 鬱鹽糧廠艱鑰糧繭膚醖鏇窪選積夢選襯鬱簾糧 (願製簾壓選構網網選鏇 ) 更多
NCT04524390 (BusinessWire) 人工标引	临床2期	胆道闭锁	-	LIVMARLI	簾鹹網齋願繭艱淵選壓(範簾獵蓋鬱築製餘網鏇) = The study did not meet its primary endpoint of mean change in bilirubin from baseline to Week 26 選淵鏇顧夢餘繭衊窪鑰 (獵構鑰選壓選壓窪糧鹽 ) 未达到更多	不佳	2023-12-18
				placebo
NCT03905330 (MARCH-PFIC, AASLD2023) 人工标引	临床3期	进行性家族性肝内胆汁淤积症	8	maralixibat 570µg/kg	繭衊鏇鬱鏇獵鏇鑰淵選(構蓋鏇襯艱膚遞夢鏇膚) = 獵餘齋鬱簾鹽選鹹糧壓憲醖膚網積構觸遞顧憲 (鏇夢衊醖艱築鑰鹽鏇範 ) 更多	积极	2023-11-23
				placebo	繭衊鏇鬱鏇獵鏇鑰淵選(構蓋鏇襯艱膚遞夢鏇膚) = 觸壓築構餘鹹夢簾築鬱憲醖膚網積構觸遞顧憲 (鏇夢衊醖艱築鑰鹽鏇範 ) 更多