Drugs / Maralixibat
Maralixibat
also known as
LUM001 · MRX · Livmarli · TAK-625 · Chlorure de maralixibat · Cloruro de maralixibat · Lopixibat · Lopixibat chloride · +3 more
Small molecule targets SLC10A2 via inhibition
Regulatory milestones approvals, filings & regulatory actions · 17 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed Alagille syndrome 5 milestones | |||||
| Filed | EU (EMA) | LIVMARLI | Alagille syndrome | 2021-09-13 | “Mirum also recently submitted a Marketing Authorization Application to the European Medicines Agency for maralixibat for the treatment of cholestatic liver disease in patients...” mirumpharma.com ↗ “Submitted marketing authorization application to the European Medicines Agency (EMA) for LIVMARLI for the treatment of cholestatic liver disease in patients with Alagille syndrome.” mirumpharma.com ↗ “Mirum has submitted a Marketing Authorization Application to the European Medicines Agency for maralixibat” mirumpharma.com ↗ “Mirum has submitted a Marketing Authorization Application to the European Medicines Agency for LIVMARLI” mirumpharma.com ↗ |
| Filed | US (FDA) | — | Alagille syndrome | 2021-02-01 | “today announced it has completed submission of its rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for maralixibat” mirumpharma.com ↗ |
| Filed | US (FDA) | — | Alagille syndrome | 2020-11-05 | “Mirum initiated its rolling New Drug Application (NDA) for the treatment of cholestatic pruritus in patients with ALGS to the U.S.” mirumpharma.com ↗ “Initiated rolling submission of New Drug Application (NDA) to U.S. Food and Drug Administration (FDA) for maralixibat” mirumpharma.com ↗ |
| Filed | US (FDA) | — | Alagille syndrome | 2020-09-01 | “Mirum has submitted a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for maralixibat for the treatment of cholestatic pruritus in patients with...” mirumpharma.com ↗ “today announced that it has submitted the first portion of its rolling New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for maralixibat.” mirumpharma.com ↗ |
| Filed | Other | LIVMARLI | Alagille syndrome | — | “Partner GC Pharma submitted a pharmaceutical approval application to the Ministry of Food and Drug Safety in Korea for LIVMARLI” mirumpharma.com ↗ |
| Filed cholestasis, progressive familial intrahepatic, 13 7 milestones | |||||
| Filed | EU (EMA) | LIVMARLI | cholestasis, progressive familial intrahepatic, 13 | 2024-04-02 | “Mirum has also submitted LIVMARLI for approval in Europe in PFIC for patients two months of age and older.” mirumpharma.com ↗ |
| Filed | China (NMPA) | LIVMARLI | cholestasis, progressive familial intrahepatic, 13 | 2023-10-17 | “the National Medical Products Administration of China (NMPA) has accepted the New Drug Application (NDA) for CAN108 (Maralixibat Chloride Oral Solution /迈芮倍®/LIVMARLI®)” canbridgepharma.com ↗ |
| Filed | US (FDA) | LIVMARLI | cholestasis, progressive familial intrahepatic, 13 | 2023-05-18 | “Mirum has submitted a supplemental new drug application to the FDA for approval of LIVMARLI in cholestatic pruritus associated with progressive familial intrahepatic cholestasis” mirumpharma.com ↗ “Mirum has also submitted LIVMARLI for approval in the U.S. (in cholestatic pruritus in PFIC patients three months of age and older) and in Europe” mirumpharma.com ↗ “Mirum has also submitted LIVMARLI for approval in the U.S. in cholestatic pruritus in PFIC patients three months of age and older, and in Europe” mirumpharma.com ↗ “Mirum has also submitted LIVMARLI for approval in the U.S. in cholestatic pruritus in PFIC patients three months of age and older and in Europe” mirumpharma.com ↗ “Mirum has also submitted LIVMARLI for approval in the U.S. in cholestatic pruritus in PFIC patients three months and older and in Europe” mirumpharma.com ↗ |
| Filed | EU (EMA) | LIVMARLI | cholestasis, progressive familial intrahepatic, 13 | 2023-04-05 | “today announced submission to the European Medicines Agency (EMA) of a variation application to the Marketing Authorization for LIVMARLI ® (maralixibat) oral solution” mirumpharma.com ↗ |
| Filed | US (FDA) | LIVMARLI | cholestasis, progressive familial intrahepatic, 13 | 2023-02-14 | “Supplemental new drug application for LIVMARLI previously submitted to U.S. FDA for treatment of cholestatic pruritus in patients with PFIC.” mirumpharma.com ↗ “Mirum has also submitted LIVMARLI for approval in the U.S. in cholestatic pruritus in PFIC patients three months of age and older” mirumpharma.com ↗ |
| Filed | EU (EMA) | — | cholestasis, progressive familial intrahepatic, 13 | 2021-03-09 | “Received validation (acceptance) of Mirum’s marketing authorization application (MAA) to the European Medicines Agency (EMA) for maralixibat for the treatment of patients with...” mirumpharma.com ↗ |
| Filed | EU (EMA) | — | cholestasis, progressive familial intrahepatic, 13 | 2020-12-09 | “Mirum has also submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for maralixibat” mirumpharma.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansions (3) | US (FDA) | LIVMARLI | — | 2023-03-13 – 2024-07-24 | fda.gov ↗ |
| Approved | EU (EMA) | Livmarli | — | 2022-12-09 | europa.eu ↗ |
| Approved | US (FDA) | LIVMARLI | — | 2021-09-29 | fda.gov ↗ |
Trials 24
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT02475317 | Jun → Dec 2015 | — | Mirum Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 214 trials · 4 met primary · 3 missed | ||||||
| Phase 2 | NCT07823127 | Jan 2027 → Jan 2029 expected | intrahepatic cholestasis of pregnancy | Mirum Pharmaceuticals, Inc. | Not yet recruiting | No outcome recorded |
| Phase 2 | NCT07389031 | Apr 2026 → Mar 2028 expected | intrahepatic cholestasis of pregnancy | Imperial College London | Not yet recruiting | No outcome recorded |
| Phase 2 | NCT04729751 | Sep 2021 → Dec 2024 | Alagille syndrome, progressive familial intrahepatic cholestasis | Mirum Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT04524390 | Jul 2021 → Nov 2023 | biliary atresia | Mirum Pharmaceuticals, Inc. | Completed | Missed primary |
| Phase 2 | NCT04168385 | Jan 2020 → Sep 2024 | cholestasis | Mirum Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT02321306 | May 2015 → Dec 2017 | primary biliary cholangitis | Mirum Pharmaceuticals, Inc. | Withdrawn | No outcome recorded Stop: Enrollment |
| Phase 2 | NCT02117713 | Mar 2015 → Jun 2020 | Alagille syndrome | Mirum Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT02057692 | Nov 2014 → Nov 2016 | Alagille syndrome | Mirum Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 2 | NCT02160782 | Oct 2014 → May 2020 | Alagille syndrome | Mirum Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 2 | NCT02057718 | Mar 2014 → May 2020 | progressive familial intrahepatic cholestasis | Mirum Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT02061540 | Mar 2014 → Feb 2016 | primary sclerosing cholangitis | Mirum Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 2 | NCT02047318 | Dec 2013 → Jun 2020 | Alagille syndrome | Mirum Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 2 | NCT01904058 | Aug 2013 → Apr 2015 | primary biliary cholangitis | Mirum Pharmaceuticals, Inc. | Completed | Missed primary |
| Phase 2 | NCT01903460 | Aug 2013 → Feb 2015 | Alagille syndrome | Mirum Pharmaceuticals, Inc. | Completed | Missed primary |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT06413368 | Apr 2025 → Jun 2027 expected | constipation disorder, cystic fibrosis | Children's Hospital Los Angeles | Recruiting | No outcome recorded |
| Phase 36 trials · 1 met primary | ||||||
| Phase 3 | NCT06553768 | Oct 2024 → Oct 2026 expected | — | Mirum Pharmaceuticals, Inc. | Active not recruiting | No outcome recorded |
| Phase 3 | NCT05543174 | Jan → Oct 2023 | Alagille syndrome | Takeda | Completed | No outcome recorded |
| Phase 3 | NCT05543187 | Jan → Sep 2023 | progressive familial intrahepatic cholestasis | Takeda | Completed | No outcome recorded |
| Phase 3 | NCT04185363 | Jan 2020 → Apr 2025 | progressive familial intrahepatic cholestasis | Mirum Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 3 | NCT03905330 | Jul 2019 → Sep 2022 | progressive familial intrahepatic cholestasis | Mirum Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 3 | NCT03353454 | Oct 2018 → Jun 2020 | progressive familial intrahepatic cholestasis | Mirum Pharmaceuticals, Inc. | Withdrawn | No outcome recorded |
| Phase 41 trial | ||||||
| Phase 4 | NCT07290257 | Sep 2025 → Sep 2030 expected | Alagille syndrome, progressive familial intrahepatic cholestasis | Mirum Pharmaceuticals, Inc. | Recruiting | No outcome recorded |
| Phase not stated1 trial | ||||||
| — | NCT04530994 | — | Alagille syndrome | Mirum Pharmaceuticals, Inc. | Approved for marketing | No outcome recorded |
News releases announcing trial results or a regulatory action · 75
| Date | Issuer | Release |
|---|---|---|
| 2026-08-18 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | Regulatory CANbridge Announces NHIA Reimbursement Approval of Livmarli ® for Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) in Taiwan canbridgepharma.com ↗
The Company today announced that Livmarli ® (maralixibat chloride oral solution), indicated for the treatment of Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC), has obtained reimbursement approval from National Health Insurance Administration (NHIA) in Taiwan. |
| 2026-05-30 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals Announces New Data from Rare Liver Disease Programs Presented at the EASL International Liver Congress 2026 mirumpharma.com ↗
Treatment with LIVMARLI® (maralixibat) demonstrates improved event-free survival in patients with progressive familial intrahepatic cholestasis (PFIC) |
| 2025-11-07 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals Showcases Leadership in Liver Disease Innovation With Data From LIVMARLI® and Volixibat Clinical Programs at AASLD’s The Liver Meeting® mirumpharma.com ↗
Maralixibat, an Ileal Bile Acid Transporter Inhibitor, Reduces Cholestatic Pruritus in PSC: Real-World Experience |
| 2025-04-14 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum’s LIVMARLI Now FDA Approved in Tablet Formulation mirumpharma.com ↗
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of LIVMARLI ® (maralixibat) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). |
| 2025-03-27 | Mirum Pharmaceuticals, Inc. | Regulatory LIVMARLI Now Approved in Japan for ALGS and PFIC mirumpharma.com ↗ |
| 2024-12-18 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | Regulatory CANbridge Announces Marketing Approval of Livmarli® in Taiwan for the Treatment of Cholestatic Pruritus in PFIC Patients canbridgepharma.com ↗
announced the marketing approval of Livmarli® (Maralixibat Chloride Oral Solution /邁芮倍®) by the Taiwan Food and Drug Administration (TFDA) for Treatment of Cholestatic Pruritus in Progressive Familial Intrahepatic Cholestasis (PFIC) patients aged 3 months and older. |
| 2024-12-18 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | Regulatory CANbridge Announces Expansion of Livmarli® Label to Include ALGS Patients as Young as 2 Months in Taiwan canbridgepharma.com ↗
This approval extends the use of Livmarli for the treatment of cholestatic pruritus in patients with ALGS to include those aged 2 months and older. |
| 2024-07-25 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum’s LIVMARLI Now Approved for PFIC in Patients 12 Months and Older mirumpharma.com ↗
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has approved a label expansion for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC). |
| 2024-07-08 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Pharmaceuticals’ LIVMARLI Approved in the European Union for Patients with PFIC mirumpharma.com ↗
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the European Commission has granted marketing authorization for LIVMARLI® (maralixibat) oral solution for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients three months of age and older. |
| 2024-06-06 | Mirum Pharmaceuticals, Inc. | Results Long-Term Data from Mirum’s LIVMARLI Studies in ALGS and PFIC Highlighted at EASL Congress mirumpharma.com ↗
Data demonstrated that nearly all patients who remained on LIVMARLI for seven years experienced benefit including clinically meaningful reductions in pruritus and serum bile acids, along with improvements in height. |
| 2024-05-31 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Pharmaceuticals Announces Positive CHMP Opinion for LIVMARLI® (maralixibat) Oral Solution for the Treatment of PFIC in Patients Three Months of Age and Older mirumpharma.com ↗
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion of LIVMARLI® (maralixibat) oral solution for the treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) in patients three months of age and older. |
| 2024-05-29 | Mirum Pharmaceuticals, Inc. | Results Long-Term Data from Mirum’s LIVMARLI Studies in ALGS and PFIC to be Presented at EASL Congress mirumpharma.com ↗
Data showcases sustained clinical benefit following 7 years of LIVMARLI treatment in patients with Alagille syndrome |
| 2024-05-21 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | Regulatory CANbridge Announces Expansion of LIVMARLI ® Label to Include Patients as Young as 3 Months in China canbridgepharma.com ↗
LIVMARLI ® (Maralixibat Chloride Oral Solution /邁芮倍 ® ) has been granted an expanded label by the National Medical Products Administration (NMPA). |
| 2024-05-18 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals’ LIVMARLI Data Showcased at ESPGHAN Annual Meeting mirumpharma.com ↗
Patients with PFIC showed significant and sustained improvements in pruritus severity, serum bile acid (sBA) levels, total bilirubin and growth following up to two years of LIVMARLI treatment. |
| 2024-05-07 | Mirum Pharmaceuticals, Inc. | Results Mirum Announces Publication of Phase 3 MARCH Data in The Lancet Demonstrating Benefits of LIVMARLI (maralixibat) in patients with PFIC mirumpharma.com ↗
The MARCH-PFIC study was the largest, randomized, double-blind, placebo-controlled study in patients with PFIC, confirming the efficacy and safety of LIVMARLI, an IBAT inhibitor, across a broad genetic range of PFIC types. |
| 2024-04-02 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Pharmaceuticals’ LIVMARLI (maralixibat oral solution) Receives Positive Reimbursement Recommendation by Canada’s CADTH for Patients with Cholestatic Pruritus in Alagille Syndrome mirumpharma.com ↗ |
| 2024-03-13 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Pharmaceuticals’ LIVMARLI Receives FDA Approval for Treatment of Cholestatic Pruritus in Patients with Progressive Familial Intrahepatic Cholestasis mirumpharma.com ↗
Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has approved LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients five years of age and older with progressive familial intrahepatic cholestasis (PFIC). |
| 2024-02-29 | Mirum Pharmaceuticals, Inc. | Results Hepatology Publishes Six-Year Data Demonstrating Improved Clinical Outcomes in Patients with Alagille Syndrome Treated with Mirum’s LIVMARLI mirumpharma.com ↗
This seminal six-year analysis of LIVMARI versus a comparable natural history control group demonstrates a 70% risk reduction for clinical outcomes in patients treated with LIVMARLI. |
| 2023-12-18 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals Announces Data from EMBARK Phase 2b Study for Biliary Atresia mirumpharma.com ↗
The study did not meet its primary endpoint of mean change in bilirubin from baseline to Week 26, or the key secondary endpoints. |
| 2023-11-13 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals Presents New Data at The Liver Meeting® mirumpharma.com ↗
Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced new data from its LIVMARLI and volixibat programs presented at The Liver Meeting®, the American Association for the Study of Liver Diseases (AASLD) annual meeting in Boston, Massachusetts. |
| 2023-10-17 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Pharmaceuticals Announces New PDUFA Date for LIVMARLI for the Treatment of Cholestatic Pruritus in Progressive Familial Intrahepatic Cholestasis mirumpharma.com ↗
Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has extended the review of the Supplemental New Drug Application (sNDA) for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC). |
| 2023-10-17 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | Regulatory CANbridge Announces NDA Acceptance of CAN108 (邁芮倍®/LIVMARLI®) for Cholestatic Pruritus in Progressive Familial Intrahepatic Cholestasis (PFIC) by China’s National Medical Products Administration canbridgepharma.com ↗
The application has been granted priority review. |
| 2023-10-09 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | Regulatory CANbridge Announces Marketing Approval of CAN108 (邁芮倍®/LIVMARLI®) in Taiwan for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome (ALGS) - LIVMARLI is the First and Only Treatment Approved in Taiwan for Cholestatic Pruritus in ALGS - LIVMARLI is approved for Cholestatic Pruritus in ALGS in the US, Canada, Europe, Mainland China, and other regions globally canbridgepharma.com ↗
CANbridge Pharmaceuticals Inc. (1228.HK) , a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies to treat rare diseases and oncology, today announced the marketing approval of CAN108 (Maralixibat Chloride Oral Solution /邁芮倍 ® /LIVMARLI®) by the Taiwan Food and Drug Administration (TFDA). |
| 2023-10-07 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals Presents LIVMARLI Clinical Data and Real-World Evidence at NASPGHAN mirumpharma.com ↗
Key data from the study demonstrated significant, rapid, and clinically meaningful improvements in pruritus and serum bile acids (sBA) along with significant improvements in bilirubin and weight z-scores. |
| 2023-09-28 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | Regulatory CANbridge Announces Marketing Approval of CAN108 (LIVMARLI ® ) in Hong Kong for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome (ALGS) - LIVMARLI is the First and Only Treatment Approved in Hong Kong for Cholestatic Pruritus in ALGS - LIVMARLI is approved for Cholestatic Pruritus in ALGS in the United States, Canada, Europe, Mainland China and other regions globally canbridgepharma.com ↗
CANbridge Pharmaceuticals Inc. (1228.HK), a global biopharmaceutical company, with a foundation in China, committed to the research, development and commercialization of transformative therapies to treat rare diseases and oncology, today announced the marketing approval of CAN108 (Maralixibat Chloride Oral Solution/LIVMARLI®) by the Pharmacy & Poisons Board of Hong Kong. |
| 2023-07-25 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum’s LIVMARLI® Now Authorized in Canada for Cholestatic Pruritus in Patients with Alagille Syndrome mirumpharma.com ↗
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that LIVMARLI® (maralixibat oral solution) has been authorized by Health Canada for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS). |
| 2023-06-23 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals Showcases Five New LIVMARLI (maralixibat) Presentations at the EASL Congress mirumpharma.com ↗
New analysis demonstrates bilirubin normalization in PFIC patients treated with LIVMARLI (maralixibat); the only IBAT inhibitor reported to show this effect |
| 2023-06-14 | Mirum Pharmaceuticals, Inc. | Results Event-Free Survival Data from Mirum’s LIVMARLI Alagille Syndrome Program Published in Hepatology mirumpharma.com ↗
The overall findings from the retrospective analysis suggest that patients with ALGS treated with LIVMARLI who meet the identified prognostic parameters may have improved six-year survival and clinical outcomes. |
| 2023-06-07 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals to Present New LIVMARLI Data at the 2023 EASL Congress mirumpharma.com ↗
Maralixibat leads to significant reductions in bilirubin for patients with Progressive Familial Intrahepatic Cholestasis |
| 2023-06-01 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | Regulatory CANbridge Announces Marketing Approval of CAN108 (迈芮倍®/LIVMARLI®) in China for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome -LIVMARLI First and Only Treatment Approved in Mainland China for Cholestatic Pruritus in ALGS -CANbridge to Hold Investor Call canbridgepharma.com ↗
announced that the Chinese National Medical Products Administration (NMPA) has approved CAN108 (Maralixibat Chloride Oral Solution /迈芮倍 ® /LIVMARLI®) to be marketed for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older. |
| 2023-05-19 | Mirum Pharmaceuticals, Inc. | Results Data from Mirum’s LIVMARLI Studies Presented at ESPGHAN Annual Meeting mirumpharma.com ↗
In ALGS, the data continue to support LIVMARLI’s safety and efficacy profile, including in infants under 12 months of age as well as in the real-world setting. |
| 2023-05-18 | Mirum Pharmaceuticals, Inc. | Results Hepatology Communications Publishes Data from Mirum’s LIVMARLI in Primary Sclerosing Cholangitis mirumpharma.com ↗
In this open-label Phase 2 pilot study, pruritus scores and serum bile acid concentration decreased over the 14 weeks of treatment with LIVMARLI. |
| 2023-05-17 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals to Showcase LIVMARLI Data at ESPGHAN Annual Meeting mirumpharma.com ↗
Maralixibat improves cholestatic pruritus and bile acids in children with FIC1: Data from the MARCH-PFIC trial |
| 2023-04-05 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Submits for European Approval of LIVMARLI in Progressive Familial Intrahepatic Cholestasis mirumpharma.com ↗
Marketing Authorization variation submitted for treatment of patients with progressive familial intrahepatic cholestasis (PFIC) two months of age and older; data supports treatment across the broadest range of genetic types ever studied. |
| 2023-03-14 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Announces Label Expansion for LIVMARLI in the United States to Include Infants Three Months of Age and Older mirumpharma.com ↗
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has approved a reduction in age from one year to three months for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS). |
| 2023-02-14 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Pharmaceuticals Submits Supplemental New Drug Application to FDA for LIVMARLI in Patients with Cholestatic Pruritus in Progressive Familial Intrahepatic Cholestasis mirumpharma.com ↗ |
| 2022-12-20 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | Regulatory CANbridge Congratulates Mirum on EU Marketing Approval for Maralixibat/LIVMARLI® to Treat Cholestatic Pruritis in Rare Liver Disease, Alagille Syndrome -Together with recent positive topline Phase 3 data in progressive familial intrahepatic cholestasis (PFIC), approval underscores maralixibat potential as promising new rare liver disease treatment -CANbridge holds exclusive Greater China license to maralixibat (CAN108) in liver diseases, China part of global trial ongoing in biliary atresia (BA） canbridgepharma.com ↗
Maralixibat is the first ALGS treatment approved in Europe. |
| 2022-12-13 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Pharmaceuticals’ LIVMARLI (maralixibat) Approved by the European Commission for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome Two Months and Older mirumpharma.com ↗
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the European Commission has granted marketing authorization for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) two months of age and older. |
| 2022-11-07 | Mirum Pharmaceuticals, Inc. | Results Mirum Presents New Data from LIVMARLI Phase 3 PFIC Study and ALGS Infant Study in Late-Breaker Presentations at The Liver Meeting of AASLD mirumpharma.com ↗
Phase 3 MARCH study meets primary endpoint and demonstrates statistical significance across key endpoints in all PFIC types. |
| 2022-10-27 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | Results CANbridge Announces Encouraging Development in Maralixibat/LIVMARLI in Rare Liver Disease - Congratulates Mirum on Positive Topline Phase 3 Data in progressive familial intrahepatic cholestasis (PFIC) - CANbridge holds exclusive Greater China license to maralixibat (CAN108) in liver diseases, China trial ongoing in biliary atresia (BA) canbridgepharma.com ↗ |
| 2022-10-24 | Mirum Pharmaceuticals, Inc. | Results Positive Topline Data Announced from Mirum’s LIVMARLI Phase 3 MARCH Study in Progressive Familial Intrahepatic Cholestasis (PFIC) mirumpharma.com ↗
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced positive topline results from its pivotal Phase 3 MARCH study evaluating LIVMARLI® (maralixibat) oral solution in 93 patients with progressive familial intrahepatic cholestasis (PFIC) in a broad range of subtypes, age one to 17 years. |
| 2022-10-18 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals’ LIVMARLI Data Highlighting Quality of Life and Sleep Improvements in Patients with Alagille Syndrome Published in Journal of Pediatrics mirumpharma.com ↗
Pruritus treatment response at week 48 was associated with clinically meaningful improvement in HRQoL from baseline to week 48. |
| 2022-10-14 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Pharmaceuticals Announces Positive CHMP Opinion for LIVMARLI® (maralixibat) Oral Solution for Cholestatic Pruritus in Patients with Alagille Syndrome Two Months of Age and Older mirumpharma.com ↗
The European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion of LIVMARLI for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) two months of age and older. |
| 2022-10-12 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals Presents Long-Term LIVMARLI® (maralixibat) Data and New Alagille Syndrome Caregiver Burden Analysis at NASPGHAN 2022 Annual Meeting mirumpharma.com ↗
Our presence at NASPGHAN provides an exciting opportunity for Mirum to showcase maralixibat six-year event-free survival benefit over the natural history standard of care, as well as long-term improvements in growth in patients with Alagille syndrome |
| 2022-10-05 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals to Present LIVMARLI® (maralixibat) Analyses and Host a Scientific Symposium at NASPGHAN 2022 Annual Meeting mirumpharma.com ↗
Poster #290: Maralixibat Improves Growth in Patients with Alagille Syndrome: A 4-Year Analysis (p. 210) |
| 2022-06-24 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals Presents New Data at The International Liver Congress™ of the European Association for the Study of the Liver (EASL) mirumpharma.com ↗
The data demonstrated that: There were 0% GI-related adverse events in the fasted state in two out of the three fasted studies. |
| 2022-06-24 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals Highlights Data Demonstrating Impact of LIVMARLI® (maralixibat) on Patients with Alagille Syndrome at European Society for Paediatric Gastroenterology, Hepatology and Nutrition (ESPGHAN) Annual Meeting mirumpharma.com ↗
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) presented data from clinical programs evaluating maralixibat (LIVMARLI®) in patients with Alagille syndrome at the 54 th Annual ESPGHAN Meeting. |
| 2022-03-29 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | Regulatory CANbridge Pharmaceuticals CAN108 New Drug Application/Orphan Drug Registration (NDA/ORD) for Alagille Syndrome Accepted by the Taiwan Food and Drug Administration canbridgepharma.com ↗ |
| 2022-01-18 | CANbridge (Suzhou) Bio-pharma Co., Ltd. | Regulatory CANbridge Pharmaceuticals CAN108 New Drug Application (NDA), for Alagille Syndrome, Accepted by China’s National Medical Products Administration Priority Review Expected to be Granted canbridgepharma.com ↗
the Chinese National Medical Products Administration (NMPA) has accepted the New Drug Application (NDA) for CAN108 (maralixibat oral solution (LIVMARLI TM )) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older. |
| 2021-12-13 | Mirum Pharmaceuticals, Inc. | Results LIVMARLI (maralixibat) Presentations Show Improvements in Quality of Life Measures in Alagille Syndrome and PFIC2 at NASPGHAN Annual Meeting 2021 mirumpharma.com ↗
Pruritus response with LIVMARLI treatment is associated with improvements in sleep disturbance in analysis of ICONIC study in Alagille syndrome. |
| 2021-12-06 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals to Present LIVMARLI™ (maralixibat) Analyses at NASPGHAN 2021 Annual Meeting mirumpharma.com ↗
Response to treatment with maralixibat in Alagille syndrome is associated with improved health-related quality of life |
| 2021-11-15 | Mirum Pharmaceuticals, Inc. | Results Six-Year Natural History Comparison with Mirum’s LIVMARLI (maralixibat) Demonstrates Event-Free Survival and Transplant-Free Survival in Patients with Alagille Syndrome mirumpharma.com ↗ |
| 2021-11-01 | Mirum Pharmaceuticals, Inc. | Results The Lancet Publishes Data From Mirum Pharmaceuticals' ICONIC Pivotal Study of Maralixibat (LIVMARLI) Treatment in Alagille Syndrome mirumpharma.com ↗
Data published in The Lancet demonstrate that maralixibat provides durable and clinically meaningful improvements across multiple disease parameters and has the potential to provide a new treatment paradigm for patients with ALGS. |
| 2021-09-29 | Mirum Pharmaceuticals, Inc. | Regulatory U.S. FDA Approves LIVMARLI (maralixibat) as the First and Only Approved Medication for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome One Year of Age and Older mirumpharma.com ↗
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leader in rare liver disease, today announced that the U.S. Food and Drug Administration (FDA) has approved LIVMARLI™ (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older. |
| 2021-09-13 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Pharmaceuticals Submits European Marketing Authorization Application for Maralixibat in Alagille Syndrome Supported by New Positive Results from Natural History Study Comparison mirumpharma.com ↗
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the submission of a Marketing Authorization Application (MAA) for the treatment of cholestatic liver disease in patients with Alagille syndrome (ALGS) to the European Medicines Agency (EMA). |
| 2021-06-23 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals Presents Analyses From Its Rare Liver Disease Programs at the EASL International Liver Congress 2021 mirumpharma.com ↗
Maralixibat was well-tolerated for more than five years. |
| 2021-06-03 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals Showcases Maralixibat Transplant-Free Survival Data for Progressive Familial Intrahepatic Cholestasis and Long-Term Safety Analyses for Alagille Syndrome during WCPGHAN Annual Meeting mirumpharma.com ↗
Safety data from more than five years of maralixibat treatment across three Phase 2 ALGS studies demonstrate that gastrointestinal events were generally mild to moderate and transient in nature, with no discontinuation of maralixibat treatment. |
| 2021-05-26 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals to Present Maralixibat Data and Host Symposium at the 6th World Congress of Pediatric Gastroenterology, Hepatology and Nutrition (WCPGHAN) Annual Meeting 2021 mirumpharma.com ↗
Five-year analysis demonstrating improvements in transplant-free survival for patients with PFIC2 featured in oral presentation; abstract nominated for the Alex Mowat Prize for best oral presentation in hepatology. |
| 2021-03-29 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Pharmaceuticals Announces FDA Acceptance of New Drug Application and Priority Review for Maralixibat in Alagille Syndrome mirumpharma.com ↗
Priority review and Rare Pediatric Disease Designation granted. |
| 2021-02-01 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Pharmaceuticals Announces Completion of Rolling NDA Submission for Maralixibat in Alagille Syndrome mirumpharma.com ↗ |
| 2021-01-14 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Pharmaceuticals Announces First Patient Enrolled in Phase 2b VISTAS Clinical Study Evaluating Volixibat in Adult Patients With Primary Sclerosing Cholangitis mirumpharma.com ↗
The company has initiated a rolling NDA submission to the U.S. Food and Drug Administration (FDA) for maralixibat for the treatment of cholestatic pruritus associated with Alagille syndrome, which Mirum expects to complete this quarter. |
| 2020-11-30 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Pharmaceuticals Announces European Medicines Agency Validation of the Marketing Authorization Application for Maralixibat in Patients with PFIC2 mirumpharma.com ↗
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced that the company’s Marketing Authorization Application (MAA) for its investigational medicine, maralixibat, for the treatment of patients with progressive familial intrahepatic cholestasis type 2 (PFIC2), also known as bile salt export pump (BSEP) deficiency, was accepted for review (validated) by the European Medicines Agency (EMA). |
| 2020-11-15 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals Announces Data Presented During AASLD Highlighting Durable Improvements in Pruritus and Quality of Life in Children with Alagille Syndrome Treated with Maralixibat mirumpharma.com ↗
Data demonstrates significant and durable improvements in pruritus in children with Alagille syndrome through 220 weeks of maralixibat treatment |
| 2020-11-13 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals Presents New Data From Its Maralixibat and Volixibat Clinical Studies at AASLD Annual Meeting mirumpharma.com ↗
Maralixibat data highlights the relationship between the reduction of serum bile acids and pruritus intensity, improved growth, and other quality of life measures. |
| 2020-11-12 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Pharmaceuticals Provides Third Quarter 2020 Financial Results and Business Update, and Announces Virtual Investor Day mirumpharma.com ↗
Initiated rolling submission of New Drug Application (NDA) to U.S. Food and Drug Administration (FDA) for maralixibat for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS). |
| 2020-09-01 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Pharmaceuticals Initiates Rolling Submission of a New Drug Application for Maralixibat for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome and Launches Expanded Access Program mirumpharma.com ↗
The Company has initiated a rolling NDA submission for maralixibat in the treatment of patients with cholestatic pruritus associated with ALGS and expects to complete the submission in the first quarter of 2021. |
| 2020-08-29 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals Presents Maralixibat Five-Year Transplant-Free Survival Data for Patients With PFIC2 at Digital International Liver Congress 2020 mirumpharma.com ↗
The five-year analysis showed that patients with PFIC2, also known as bile salt export pump (BSEP) deficiency, who achieved sBA control on long-term maralixibat treatment have a significant improvement in transplant-free survival. |
| 2020-06-02 | Mirum Pharmaceuticals, Inc. | Results Mirum’s Maralixibat Demonstrates Transplant-Free Survival for Pediatric Patients with Progressive Familial Intrahepatic Cholestasis mirumpharma.com ↗
The data demonstrated that patients with long-term maralixibat treatment who achieved serum bile acid (sBA) control, have five-year transplant-free survival after treatment. |
| 2019-11-08 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals Presents Data Demonstrating Long-term Durability of Treatment Effect of Maralixibat in Children With Cholestatic Liver Diseases mirumpharma.com ↗
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced that results from the long-term extension of the Phase 2b ICONIC study demonstrate the durability of treatment effect and disease-modifying potential of maralixibat in children with Alagille syndrome (ALGS). |
| 2019-10-28 | Mirum Pharmaceuticals, Inc. | Regulatory Mirum Pharmaceuticals Announces Breakthrough Therapy Designation for Maralixibat for the Treatment of Pruritus Associated with Alagille Syndrome mirumpharma.com ↗
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for maralixibat for the treatment of pruritus associated with Alagille syndrome (ALGS) in patients 1 year of age and older. |
| 2019-10-21 | Mirum Pharmaceuticals, Inc. | Results Durability of Treatment Effect of Mirum Pharmaceuticals’ Maralixibat for Children With Alagille Syndrome Featured in Late Breaking Session at the Liver Meeting 2019 mirumpharma.com ↗
In ICONIC, long-term treatment with maralixibat was associated with durability of treatment response on pruritus measures as well as serum bile acids and xanthomas. |
| 2019-04-15 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals Presents New Data Demonstrating Durable Improvements in Clinical Outcome Measures in Patients with PFIC2 and Alagille Syndrome Treated with Maralixibat mirumpharma.com ↗
In the Phase 2 ICONIC study in children with Alagille syndrome, maralixibat treatment led to profound and durable improvement in pruritis, xanthomas (lipid accumulation in the skin) and bile acids over 48 weeks. |
| 2019-03-27 | Mirum Pharmaceuticals, Inc. | Results Mirum Pharmaceuticals to Present Phase 2 Clinical Results of Maralixibat in Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis at the International Liver Congress mirumpharma.com ↗
Maralixibat shows reductions in pruritus and serum bile acids in 48 week analysis of ICONIC Phase 2b study in Alagille syndrome patients |
| 2016-06-13 | Takeda | Regulatory Shire Receives FDA Breakthrough Therapy Designation for SHP621 and SHP625, Investigational Products for Rare Gastrointestinal Conditions takeda.com ↗
Shire plc (LSE: SHP, NASDAQ: SHPG) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for two investigational products for rare diseases: SHP621 (budesonide oral suspension, or BOS) for eosinophilic esophagitis (EoE), and SHP625 (maralixibat) for progressive familial intrahepatic cholestasis type 2 (PFIC2). |
| 2015-04-09 | Takeda | Results Shire Reports Topline Results from Phase 2 Studies in Children with Alagille Syndrome takeda.com ↗
Mean serum bile acid levels and pruritus at the end of the study were lower in both SHP625 and placebo treated groups as compared to baseline. |
All press releases naming this drug 95 releases
Evidence & citations 16 cited values
Every value below carries the sentence it was read from. 25 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Maralixibat | “LUM001, also known as Maralixibat (MRX)” NCT02057718 ↗15“LUM001 also known as Maralixibat (MRX)” NCT02047318 ↗ |
| Known as | Chlorure de maralixibat | ChEMBL registry synonym — accepted as the source's own label CHEMBL363392 ↗ |
| Known as | Cloruro de maralixibat | ChEMBL registry synonym — accepted as the source's own label CHEMBL363392 ↗ |
| Known as | Livmarli | “Maralixibat 9.5 MG/ML [Livmarli]” NCT06413368 ↗ |
| Known as | Lopixibat | ChEMBL registry synonym — accepted as the source's own label CHEMBL363392 ↗ |
| Known as | Lopixibat chloride | ChEMBL registry synonym — accepted as the source's own label CHEMBL363392 ↗ |
| Known as | LUM-001 CATION | ChEMBL registry synonym — accepted as the source's own label CHEMBL363392 ↗ |
| Known as | LUM001 | “Participant will receive LUM001 also known as Maralixibat (MRX) administered orally once per day.” NCT02117713 ↗8“LUM001, also known as Maralixibat (MRX) will be administered orally Once Daily (OD).” NCT02160782 ↗ |
| Known as | Maralixibat cation | ChEMBL registry synonym — accepted as the source's own label CHEMBL363392 ↗ |
| Known as | MARALIXIBAT CHLORIDE | ChEMBL registry synonym — accepted as the source's own label CHEMBL363392 ↗ |
| Known as | MRX | “Participant will receive LUM001 also known as Maralixibat (MRX) administered orally once per day.” NCT02117713 ↗4“Evaluate the long-term safety of maralixibat (MRX) in subjects with cholestatic liver disease” NCT04168385 ↗ “LUM001, also known as Maralixibat (MRX) will be administered orally Once Daily (OD).” NCT02160782 ↗ “LUM001, also known as Maralixibat (MRX)” NCT02057718 ↗ “LUM001 also known as Maralixibat (MRX)” NCT02047318 ↗ |
| Known as | TAK-625 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05543187 ↗ |
| Action | Inhibit | “an Apical Sodium-dependent Bile Acid Transporter Inhibitor (ASBTi)” NCT01903460 ↗1“Ileal bile acid transporter inhibitor” CHEMBL363392 ↗ |
| Modality | Small molecule | “A small molecule inhibitor of the ileal bile acid transporter (IBAT)” NCT04524390 ↗ |
| Route | Oral | “LUM001 administered orally once each day” NCT01903460 ↗ |
| Target | SLC10A2 | “Ileal bile acid transporter inhibitor” CHEMBL363392 ↗ |