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2024-10-01

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

NCT06193928 / RecruitingN/A

Long-Term Safety and Clinical Outcomes of Livmarli in Patients With Alagille Syndrome (LEAP)

The goal of this observational study is to evaluate the long-term safety and clinical outcomes of Livmarli prescribed to patients with Alagille Syndrome (ALGS).

开始日期2023-09-21

申办/合作机构

Mirum Pharmaceuticals, Inc.

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专利（医药）

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项与氯马昔巴特相关的文献（医药）

2024-10-04·JOURNAL OF ORGANIC CHEMISTRY

Stereoselective Synthesis of Maralixibat via VO(acac)₂/Schiff Base-Catalyzed Asymmetric Oxidation of Its Sulfide Intermediate

Article

作者: Peng, Ziyu ; Mpofo, Mercy ; Ye, Long ; Jie, Zheng

The stereoselective synthesis of maralixibat was achieved by harnessing the chiral transferring effect of the stereogenic R-sulfoxide functionality, which was obtained via the VO(acac)₂/Schiff base-catalyzed asymmetric oxidation of a phenylthiophenol prochiral intermediate. The R-sulfoxide intermediate underwent a ring closure reaction to form the seven-membered ring core structure with the desired stereochemistry, ultimately ensuring the drug's exceptional isomeric purity and synthetic efficiency.

2024-09-20·Zhonghua gan zang bing za zhi = Zhonghua ganzangbing zazhi = Chinese journal of hepatology

[Progress in the treatment of progressive familial intrahepatic cholestasis].

Review

作者: Liu, T ; Wang, J S

Progressive familial intrahepatic cholestasis (PFIC) is an important cause of liver-related death or transplantation in children. The PFIC spectrum is expanding, twelve types of PFIC are currently included in the Online Mendelian Inheritance in Man (OMIM) database. With the increase of PFIC types and the inconsistence of certain types in numbering, the current numbering classification of PFIC is confusing, so the experts in the field recommend using the corresponding mutant gene/ protein defect to name different type of PFIC except for PFIC type 1-3. The clarification of the genotype-phenotype relationship and/or the establishment of phenotypic predictors significantly improved the management of patients with PFIC. Odevixibat and maralixibat, inhibitors of the apical sodium ion-dependent bile acid transporter on the intestinal epithelial cells, were approved in European Union and the United States for the treatment of PFIC pruritus in 2021, expanding the treatment options for PFIC. Additionally, personalized treatments for specific mutations and novel gene therapy is promising.

2024-07-01·Lancet Gastroenterology & Hepatology

Maralixibat in progressive familial intrahepatic cholestasis (MARCH-PFIC): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

Article

作者: Laverdure, Noemie ; Miethke, Alexander G ; Ovchinsky, Nadia ; Nunes, Tiago ; Arikan, Cigdem ; Ordonez, Felipe ; Mittal, Naveen ; Thompson, Richard J ; Gilmour, Susan ; Longpre, Lara ; D'Agostino, Daniel ; Hupertz, Vera F ; D'Antiga, Lorenzo ; Moukarzel, Adib ; Sokal, Etienne ; Ekong, Udeme ; Squires, Robert H ; Chiou, Fang Kuan ; Porta, Gilda ; Vig, Pamela ; Laborde, Nolwenn ; Baumann, Ulrich ; Horslen, Simon P ; Mosca, Antonella ; Lascau, Anamaria ; Gonzalez-Peralta, Regino P ; Garner, Will ; Czubkowski, Piotr ; Jaecklin, Thomas ; Covarrubias Esquer, Joshue ; Aqul, Amal A ; Mogul, Douglas B ; Hartley, Jane ; Huber, Wolf-Dietrich ; Kasi, Nagraj ; Lin, Chuan-Hao

BACKGROUND:

Progressive familial intrahepatic cholestasis (PFIC) is a group of autosomal recessive disorders, the most prevalent being BSEP deficiency, resulting in disrupted bile formation, cholestasis, and pruritus. Building on a previous phase 2 study, we aimed to evaluate the efficacy and safety of maralixibat-an ileal bile acid transporter inhibitor-in participants with all types of PFIC.

METHODS:

MARCH-PFIC was a multicentre, randomised, double-blind, placebo-controlled, phase 3 study conducted in 29 community and hospital centres across 16 countries in Europe, the Americas, and Asia. We recruited participants aged 1-17 years with PFIC with persistent pruritus (>6 months; average of ≥1·5 on morning Itch-Reported Outcome [Observer; ItchRO(Obs)] during the last 4 weeks of screening) and biochemical abnormalities or pathological evidence of progressive liver disease, or both. We defined three analysis cohorts. The BSEP (or primary) cohort included only those with biallelic, non-truncated BSEP deficiency without low or fluctuating serum bile acids or previous biliary surgery. The all-PFIC cohort combined the BSEP cohort with participants with biallelic FIC1, MDR3, TJP2, or MYO5B deficiencies without previous surgery but regardless of bile acids. The full cohort had no exclusions. Participants were randomly assigned (1:1) to receive oral maralixibat (starting dose 142·5 μg/kg, then escalated to 570 μg/kg) or placebo twice daily for 26 weeks. The primary endpoint was the mean change in average morning ItchRO(Obs) severity score between baseline and weeks 15-26 in the BSEP cohort. The key secondary efficacy endpoint was the mean change in total serum bile acids between baseline and the average of weeks 18, 22, and 26 in the BSEP cohort. Efficacy analyses were done in the intention-to-treat population (all those randomly assigned) and safety analyses were done in all participants who received at least one dose of study drug. This completed trial is registered with ClinicalTrials.gov, NCT03905330, and EudraCT, 2019-001211-22.

FINDINGS:

Between July 9, 2019, and March 4, 2022, 125 patients were screened, of whom 93 were randomly assigned to maralixibat (n=47; 14 in the BSEP cohort and 33 in the all-PFIC cohort) or placebo (n=46; 17 in the BSEP cohort and 31 in the all-PFIC cohort), received at least one dose of study drug, and were included in the intention-to-treat and safety populations. The median age was 3·0 years (IQR 2·0-7·0) and 51 (55%) of 93 participants were female and 42 (45%) were male. In the BSEP cohort, least-squares mean change from baseline in morning ItchRO(Obs) was -1·7 (95% CI -2·3 to -1·2) with maralixibat versus -0·6 (-1·1 to -0·1) with placebo, with a significant between-group difference of -1·1 (95% CI -1·8 to -0·3; p=0·0063). Least-squares mean change from baseline in total serum bile acids was -176 μmol/L (95% CI -257 to -94) for maralixibat versus 11 μmol/L (-58 to 80) for placebo, also representing a significant difference of -187 μmol/L (95% CI -293 to -80; p=0·0013). The most common adverse event was diarrhoea (27 [57%] of 47 patients on maralixibat vs nine [20%] of 46 patients on placebo; all mild or moderate and mostly transient). There were five (11%) participants with serious treatment-emergent adverse events in the maralixibat group versus three (7%) in the placebo group. No treatment-related deaths occurred.

INTERPRETATION:

Maralixibat improved pruritus and predictors of native liver survival in PFIC (eg, serum bile acids). Maralixibat represents a non-surgical, pharmacological option to interrupt the enterohepatic circulation and improve the standard of care in patients with PFIC.

FUNDING:

Mirum Pharmaceuticals.

212

项与氯马昔巴特相关的新闻（医药）

2024-11-14

·Business Wire

Mirum Pharmaceuticals to Showcase Data from its LIVMARLI and Volixibat Clinical Programs at AASLD’s The Liver Meeting

FOSTER CITY, Calif.--( BUSINESS WIRE )--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that it will present data at the American Association for the Study of Liver Disease’s (AASLD) The Liver Meeting®, taking place November 15-19, 2024, in San Diego, California. Enclosed below are the titles that have been accepted for presentation during the meeting. The abstracts are available via the AASLD website . Full analyses will be available following their presentation within the Publications & Presentation section on Mirum’s website . Presentations Abstract #5038: Volixibat for Cholestatic Pruritus in Primary Biliary Cholangitis: An Adaptive, Randomized, Placebo-controlled Phase 2b Trial (VANTAGE): Interim Results **Late-breaker poster presentation** Monday, November 18 from 1:00-2:00pm, Poster Hall C Presented by Dr. Kris Kowdley, Washington State University, Seattle, Washington, USA Abstract #184: Improvements in Pruritus are Associated with Improvements in Growth in Patients with Progressive Familial Intrahepatic Cholestasis: Data from the MARCH-ON trial **Oral presentation** Sunday, November 17 from 12:15-12:30pm during the ‘Advances in Pediatric Liver Disease’ session, Ballroom 6C Presented by Dr. Alexander Miethke, Cincinnati Children’s Hospital, Cincinnati, Ohio, USA Abstract #4347: Pilot Study of Volixibat Co-administered with OCA for Primary Biliary Cholangitis (PBC) Treatment: The VLX-602 Trial Monday, November 18 from 1:00-2:00pm during Poster Session IV, Poster Hall C Presented by Dr. Kris Kowdley, Washington State University, Seattle, Washington, USA Abstract #4400: Impact of Maralixibat on Caregiver Burden for Patients with Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis Monday, November 18 from 1:00-2:00pm during Poster Session IV, Poster Hall C Presented by Dr. Natasha Dilwali, The Johns Hopkins School of Medicine, Baltimore, Maryland, USA Abstract #4419 : Real-world Use of Maralixibat in Biliary Atresia: A Case Series Monday, November 18 from 1:00-2:00pm during Poster Session IV, Poster Hall C Presented by Dr. Natasha Dilwali, The Johns Hopkins School of Medicine, Baltimore, Maryland, USA Abstract #4432: The Relationship Between Serum Bile Acids and Event-Free Survival Following the Use of Maralixibat for Progressive Familial Intrahepatic Cholestasis: Data from MARCH/MARCH-ON Monday, November 18 from 1:00-2:00pm during Poster Session IV, Poster Hall C Presented by Professor Richard Thompson, King’s College London, United Kingdom Abstract #4436: Bile Acid Subspecies are Correlated with Pruritus and Bilirubin Improvement in PFIC Patients Treated with Maralixibat: Data from MARCH and MARCH-ON Monday, November 18 from 1:00-2:00pm during Poster Session IV, Poster Hall C Presented by Professor Henkjan Verkade, University of Groningen, Groningen, Netherlands Alagille Syndrome Patient & Physician Fireside Discussion During the meeting, Mirum will host a session spotlighting a pediatric hepatologist and an adult patient living with ALGS. Dr. Ajay Jain, St. Louis University, will discuss clinical insights and patient, Emma, will share her experience with LIVMARLI. Sunday, November 17 from 1:30-2:00 p.m. in Exhibit Hall TLM Theater #2 About LIVMARLI® (maralixibat) oral solution LIVMARLI® (maralixibat) oral solution 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 Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS and PFIC. 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 Alagille syndrome 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 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 , CHOLBAM® (cholic acid) capsules, and CHENODAL® (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 also initiating 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 or liver disease. CHENODAL has received medical necessity recognition by the FDA to treat patients with cerebrotendinous xanthomatosis (CTX). Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver 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. Lastly, chenodiol, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023. Mirum has submitted a new drug application with the FDA for the approval of chenodiol to treat CTX in the U.S. To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook , LinkedIn , Instagram and Twitter (X). Forward-Looking Statements This press release includes forward-looking statements pertaining to the Company’s planned participation at a scientific conference, including data presentation title and synopsis for both commercial and clinical programs, which may include discussion of the Company’s clinical and research data relating to the therapeutic potential and/or commercial viability of LIVMARLI or other Company product candidates in various liver disease indications and in patient populations that are investigational only. Words such as “will,” “could,” “would,” “potential” 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 and the other risks described in Mirum’s filings with the Securities and Exchange Commission. 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. A further description of risks and uncertainties can be found in Mirum’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors,” as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov .

临床2期突破性疗法上市批准孤儿药临床3期

2024-11-13

·北海康成

北海康成治疗戈谢病的CAN103上市申请（NDA）获国家药监局（NMPA）正式受理

中国苏州，2024年11月13日—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。公司今日宣布，中国国家药品监督管理局（NMPA）已正式受理CAN103（注射用维拉苷酶β）用于治疗成人和儿童I型和III型戈谢病的新药申请（NDA）。 CAN103是北海康成与药明生物（2269.HK）在罕见病领域的首个合作项目，是中国首个针对戈谢病开发的酶替代疗法（ERT）。据弗若斯特沙利文统计，2020年中国约有3,000名戈谢病患者。国家药监局正式受理CAN103的新药上市申请是我们取得的又一重要里程碑。获批后， CAN103将是我国首个本土自主研发并进入临床应用的戈谢病酶替代疗法药物 , 属一类创新药并可完全替代同类进口产品。同时，CAN103在全链条研创过程中积累了完整的研发数据和成本数据，将为打通本土罕见病新药研发商业化闭环提供首个案例，最终满足众多患者多年来的治疗需求。我们预计在2025年将CAN103推向市场，这将是继海芮思和迈芮倍之后北海康成上市的第三个罕见病治疗药物，也是北海康成将进入商业化的首个自主研发产品。北海康成于今年 8 月公布了 CAN103 针对成人和儿童戈谢病患者关键临床试验的积极顶线数据结果。国家药品监督管理局药品审评中心（CDE）于 9 月授予了 CAN103 优先审评资格。今年 10 月，北海康成顺利通过药品上市许可持有人（MAH）的生产和质量管理体系审核和现场检查，获得《药品生产许可证》B证。 2024年10月22日，国家药监局正式印发《生物制品分段生产试点工作方案》。在北海康成与药明生物的紧密合作下，双方顺利取得了相关生物药制品生产许可证，成为中国首批MAH制度下生物制品分段生产试点的实践者。 CAN103（注射用维拉苷酶β）是国内首个本土自主研发并即将进入临床应用的用于治疗I型和III型戈谢病患者的重组人源脑苷脂酶替代疗法。大多数戈谢病患者为I型和III型，分别为慢性非神经病变型和慢性神经病变型。CAN103通过静脉输注特异性地补充戈谢病患者体内溶酶体中缺乏的葡萄糖脑苷脂酶。 CAN103 的关键性临床试验于2024年8月取得积极顶线数据，是一项随机、双盲、剂量比较研究，旨在评估静脉滴注CAN103每2周一次在初治戈谢病患者中的有效性、安全性和药代动力学，并设有开放标签的扩展期。结果表明，该研究在60U/kg剂量组（P<0.0001）和较低的30U/kg剂量组（P<0.001）都成功达到了其主要疗效终点，即治疗9个月后受试者脾脏体积较基线的平均缩小百分比。该研究方案主要终点的设定已获得国家药品监督管理局药品审评中心(CDE)认可。戈谢病是一种最常见的溶酶体贮积症之一，为位于1q22号染色体长臂上的葡萄糖脑苷脂酶基因突变引起的常染色体隐性遗传病。该病对男性和女性的影响是同等的。临床上戈谢病包括围产期致死型、I型（非神经病变型）、II型（急性神经病变型）和III型（慢性神经病变型），其中I型和III型戈谢病患者多能活到成年。戈谢病是由葡萄糖脑苷脂酶（酸性β-葡萄糖苷酶）缺乏引起的，这种酶有助于分解溶酶体内一种被称作葡萄糖脑苷脂（葡糖神经酰胺）的细胞膜鞘脂。葡萄糖脑苷脂主要积聚在某些器官内的单核巨噬细胞系细胞中（Gaucher细胞），导致脾肿大，肝肿大，贫血，血小板减少症，骨痛和骨折，严重的戈谢病类型（围产期致死型，II型和III型），早期可出现神经系统症状。30年以来，重组人源葡萄糖脑苷脂酶替代疗法 (ERT) 一直是戈谢病的标准治疗方法，临床试验和真实世界数据表明，患者的主要非神经系统症状和体征以及生活质量得到了显着改善。根据弗若斯特沙利文的报告数据显示，2020年在中国约有3000名戈谢病患者。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11个具有可观市场潜力的药物资产组合，其中包括 3 个已获批上市产品和 8 个在研药物。这些产品均针对一些较普遍的罕见疾病和罕见肿瘤适应症，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

申请上市优先审批临床3期

2024-10-18

·北海康成

全球创新药研讨会推动京琼两地医保、医疗、医药领域深度融合！

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。近日，2024年度全球创新药研讨会暨北京普惠健康保特药及国谈药“双通道”药品培训会开幕。会议由北京医疗保险协会、海南乐城医商融合研究发展中心共同主办。40多家北京医疗机构200多位知名医学专家汇聚一堂，研讨推进全球创新药发展成果及应用，推进北京普惠健康保特药及国谈药“双通道”药品落地使用，共同书写京琼两地高水平开放、高质量发展新篇章。本次会议的举办，旨在将北京市的医疗资源优势与海南自贸港的“先行先试”政策优势有机结合起来，共同推动北京普惠健康保海内外特药和国谈药“双通道”药品落地使用，助力发展创新医药新质生产力，让更多、更新全球创新药惠及北京市乃至全国参保患者，服务国家发展战略和国民健康需求，推动京琼两地多层次医疗保障体系高质量发展。北京市医保局党组书记、局长马继业发表开幕致辞。他表示，北京的医疗资源、医药健康科研资源得天独厚，乐城先行区则拥有“先行先试”的政策优势。此次大会的成功召开，将进一步增强医生对全球创新药的全面深入了解，加速创新药品的临床应用；也将把北京的资源优势和海南的政策优势有机结合起来，发挥1+1大于2的功效，让人民群众共享改革成果。未来，北京市医保局将继续与海南省医保局、乐城先行管理区保持良好的长效合作机制，深化京琼两地创新医药领域务实合作，携手助力两地新质生产力发展，共同书写高水平开放、高质量发展的时代新篇章。海南省医疗保障局党组书记、局长李文秀表示，解决居民就医看病的可及性和可支付性，要大力发展多层次医疗保障体系。海南通过推进“双通道”药品管理政策落地，并联合乐城管理局共同指导推出乐城特药险，将自付药，尤其是海外创新药纳入保障责任，并不断扩展特药覆盖疾病种类，不仅提高了居民使用国谈药的可及性，也为居民享受与国际同步的药械提供可支付解决方案。乐城先行区管理局党委书记、局长贾宁表示，得益于“先行先试”政策优势，乐城已成为全球创新药械进入中国市场的“国际窗口”，已落地特许药械数量超430种，涵盖癌症肿瘤、罕见病等“救命药”，以及眼科、内分泌、医美等提高生活品质的创新产品。同时，通过检查结果互认和随访数据共享，乐城园区医院正积极与全国其他地区医院合作，使更多患者能更便捷地使用特许药械，并与合作医院及医生团队共享创新药械临床使用的科研成果。首都儿科研究所附属儿童医院消化内科王美娟医师分享用于治疗3月龄及以上阿拉杰里综合征（ALGS）患者胆汁淤积性瘙痒的迈芮倍®（LIVMARLI®/氯马昔巴特口服溶液）患者用药案例。同时，该理赔案例也在开幕式上进行了汇报分享。近年来，为持续完善多层次医疗保障体系，满足人民群众医疗保障需求，北京市医保局指导推出了北京普惠健康保，运行三年，参保人数逐年稳步增长，2024年度参保人数达425万人。北京普惠健康保每年为参保人提供100余种国内外特药保障，并建立特药清单动态调整机制，结合国家医保药品目录调整、创新药上市等情况，动态调整特药保障范围，不断满足群众用药需求。据了解，北京普惠健康保特药覆盖了肺癌、胃癌、乳腺癌、淋巴瘤等近40种肿瘤的全周期靶向用药。目前，特药保障共114种药品，其中，49种国内特药为国内医保目录外特药；65种国外特药为未在国内上市但可在博鳌乐城先行区使用的全球创新药，保障患者不出国门就能用上全球创新药。本次会议主要聚焦2024年度北京普惠健康保特药和国谈药“双通道”药品开设多个分论坛，重点培训肺癌、血液、甲乳、消化、肝胆、泌尿生殖、神经、心血管等领域50余场创新药品，结合分享药品使用案例，实地参观调研博鳌乐城医疗机构，进一步加强医生对创新药的全面深入认识，深化京琼两地医保、医疗、医药领域的深度融合，推动两地多层次医疗保障体系高质量发展迈上新台阶。中国医科院肿瘤医院、北京清华长庚医院、北京大学人民医院等40多家北京顶级医院的知名专家，北京市医保局、海南省医保局、乐城管理局主要领导，北京普惠健康保主承保公司主要领导出席研讨会。文章来源：乐成先行区管理局发布（略有修改）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12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9个在研药物。这些产品均针对一些较普遍的罕见疾病适应症，比如亨特氏综合征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、脊髓性肌萎缩症（SMA）、杜氏肌肉营养不良症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、UMass Chan医学院、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

申请上市

100 项与氯马昔巴特相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB16226

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

未上市

10 条进展最快的记录,

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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.	2024-10-01
胆汁淤积性肝病	临床3期	巴西	Mirum Pharmaceuticals, Inc.	2024-10-01
胆汁淤积性肝病	临床3期	法国	Mirum Pharmaceuticals, Inc.	2024-10-01
胆汁淤积性肝病	临床3期	德国	Mirum Pharmaceuticals, Inc.	2024-10-01
胆汁淤积性肝病	临床3期	意大利	Mirum Pharmaceuticals, Inc.	2024-10-01
胆汁淤积性肝病	临床3期	黎巴嫩	Mirum Pharmaceuticals, Inc.	2024-10-01
胆汁淤积性肝病	临床3期	墨西哥	Mirum Pharmaceuticals, Inc.	2024-10-01
胆汁淤积性肝病	临床3期	波兰	Mirum Pharmaceuticals, Inc.	2024-10-01
胆汁淤积性肝病	临床3期	西班牙	Mirum Pharmaceuticals, Inc.	2024-10-01
胆汁淤积性肝病	临床3期	英国	Mirum Pharmaceuticals, Inc.	2024-10-01

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临床结果

适应症

分期

评价

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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


NCT05543174 (CTgov)	临床3期	阿拉吉欧综合症	7	TAK-625	鹹膚艱鹽鏇蓋艱選積鏇(築糧選壓膚鹹積鏇淵願) = 構壓鬱願鏇鹽築壓願膚夢築遞艱積鑰顧餘膚鹹 (壓艱構糧鏇鹽鹽願蓋憲, 遞鏇簾積積窪願願憲窪 ~ 醖鬱夢衊鹽構遞鬱艱積) 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)	餘鏇鑰簾積願糧製餘觸(淵鹹醖觸積窪積獵窪壓) = 糧製積範齋憲鹽選夢蓋齋積淵淵觸廠廠艱鑰鑰 (壓鹹遞範廠鑰積糧襯積, 淵遞鬱鏇鹹繭壓壓膚鬱 ~ 築窪築齋齋積觸觸夢獵) 更多	-	2024-09-24
				TAK-625 (Supplemental Cohort: TAK-625)	餘鏇鑰簾積願糧製餘觸(淵鹹醖觸積窪積獵窪壓) = 襯膚願觸艱蓋憲壓蓋廠齋積淵淵觸廠廠艱鑰鑰 (壓鹹遞範廠鑰積糧襯積, 襯築淵衊積醖膚壓簾憲 ~ 蓋夢膚廠簾壓廠鬱襯艱) 更多
MARCH-ON (Biospace) 人工标引	N/A	进行性家族性肝内胆汁淤积症	-	LIVMARLI	憲築醖選糧觸艱製觸齋(鏇觸鏇壓夢積繭憲醖範) = 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. 鏇繭範選鹽遞鹽淵窪範 (鹹鬱範廠鏇齋膚衊積餘 )	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	鏇觸觸夢齋築製獵窪鑰(獵蓋壓遞醖淵衊願壓蓋) = 廠鹹淵鬱淵鬱壓鹹製鑰窪壓壓遞窪選餘觸網糧 (積膚築壓顧築積鹽構膚 ) 更多	积极	2024-05-06
				Placebo	觸築餘繭壓夢觸願遞糧(鹽築鑰範觸窪夢築壓願) = 醖鑰積淵獵簾夢憲窪積艱淵淵衊繭餘築廠選鬱 (廠鹹觸構夢廠壓壓觸壓 ) 更多
EMBARK (BusinessWire) 人工标引	临床2期	胆道闭锁	-	LIVMARLI	淵鬱遞願襯範顧築鏇淵(鬱鹹鑰鹹簾選衊遞觸選) = The study did not meet its primary endpoint of mean change in bilirubin from baseline to Week 26 鹹齋艱餘觸鹽淵艱艱糧 (願艱膚鏇鏇艱鹹網憲醖 ) 未达到更多	不佳	2023-12-18
				placebo
MARCH (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	範蓋鏇窪衊淵膚艱壓膚(簾選餘鏇製鏇鬱淵鑰夢) = 簾廠壓衊齋醖廠艱願淵積願鹽製網蓋顧願積鹽 (簾顧壓遞簾遞觸膚糧製 ) 更多	积极	2023-11-23
				placebo	範蓋鏇窪衊淵膚艱壓膚(簾選餘鏇製鏇鬱淵鑰夢) = 鏇窪淵餘鬱獵獵壓糧鹹積願鹽製網蓋顧願積鹽 (簾顧壓遞簾遞觸膚糧製 ) 更多
MARCH-PFIC (AASLD2023) 人工标引	临床3期	进行性家族性肝内胆汁淤积症	11	maralixibat (TJP2 deficiency)	膚廠鏇觸網淵鏇窪憲憲(餘淵壓糧繭衊衊壓窪窪) = Safety signals were consistent with other PFIC types. 鑰鬱選憲鬱遞餘鑰夢餘 (膚鬱衊鹽製蓋簾餘艱夢 ) 更多	积极	2023-11-13
				placebo (TJP2 deficiency)
MARCH-PFIC (AASLD2023) 人工标引	临床3期	进行性家族性肝内胆汁淤积症	64	maralixibat	衊積願夢蓋夢蓋鏇壓鏇(餘顧積窪蓋繭網鑰獵構) = 淵網襯築獵蓋糧廠繭壓願膚獵積襯獵憲膚簾廠 (鑰襯獵襯觸製憲衊構醖 )	积极	2023-11-13
				placebo	衊積願夢蓋夢蓋鏇壓鏇(餘顧積窪蓋繭網鑰獵構) = 蓋餘築鏇餘獵鏇獵範選願膚獵積襯獵憲膚簾廠 (鑰襯獵襯觸製憲衊構醖 )
AASLD2023	N/A	关节软化-肾功能不全-胆汁淤积综合征	-	Maralixibat	鏇網齋鹹廠窪艱齋範遞(鹽範鏇壓網蓋鹽積鏇觸) = 齋願繭簾製獵衊衊齋淵觸夢齋範窪獵觸艱繭夢 (鹽網簾膚膚餘鹽醖鑰鬱 ) 更多	-	2023-11-10