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 · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| 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 23 · a red edge is where a trial was stopped
| 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 213 trials | ||||||
| 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 | No outcome recorded |
| 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| Phase 2 | NCT02321306 | May 2015 → Dec 2017 | primary biliary cholangitis | Mirum Pharmaceuticals, Inc. | Withdrawn | No outcome recorded |
| 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 | No outcome recorded |
| Phase 2 | NCT02160782 | Oct 2014 → May 2020 | Alagille syndrome | Mirum Pharmaceuticals, Inc. | Completed | No outcome recorded |
| 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 | No outcome recorded |
| Phase 2 | NCT02047318 | Dec 2013 → Jun 2020 | Alagille syndrome | Mirum Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT01903460 | Aug 2013 → Feb 2015 | Alagille syndrome | Mirum Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT01904058 | Aug 2013 → Apr 2015 | primary biliary cholangitis | Mirum Pharmaceuticals, Inc. | Completed | No outcome recorded |
| 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 | ||||||
| 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 | No outcome recorded |
| 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 |
Evidence & citations 16 cited values
Every value below carries the sentence it was read from. 24 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Maralixibat | “LUM001, also known as Maralixibat (MRX)” NCT02057718 ↗ |
| 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 ↗ |