Product Description
Maralixibat (SHP625; formerly LUM001 or lopixibat) is a potent, apical, sodium‐dependent, bile acid transporter competitive inhibitor with minimal systemic absorption. Maralixibat works by reducing systemic levels of bile acids. (Sourced from: https://drugs.ncats.io/drug/V78M04F0XC)
Mechanisms of Action: IBAT Inhibitor
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: None *
Approval Status: Approved
Approved Countries: Austria | Belgium | Croatia | Czech | European Medicines Agency | Finland | France | Germany | Hungary | Iceland | Ireland | Israel | Italy | Korea | Latvia | Lithuania | Poland | Portugal | Romania | Slovakia | Slovenia | Spain | Sweden | United States
Approved Indications: Alagille Syndrome | Pruritus
Known Adverse Events: Abdominal Pain | Pain Unspecified | Vitamin D Deficiency | Diarrhea | Injuries/wounds Unspecified
Company: Mirum Pharmaceuticals
Company Location: FOSTER CITY CA 94404
Company CEO: Christopher Peetz
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Argentina, Australia, Austria, Belgium, Brazil, Canada, China, Colombia, France, Germany, Hungary, Italy, Japan, Lebanon, Mexico, Poland, Singapore, Spain, Taiwan, Turkey, United Kingdom, United States, Vietnam
Active Clinical Trial Count: 14
Highest Development Phases
Phase 3: Alagille Syndrome|Cholestasis|Cholestasis, Intrahepatic|Cholestatic Liver Disease|Pruritus
Phase 2: Biliary Atresia|Biliary Cirrhosis|Hypertension, Portal|Liver Cirrhosis|Liver Transplant
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| MRX-802 | P3 |
Unknown Status |
Pruritus |
2027-02-26 |
|
| EXPAND | P3 |
Recruiting |
Pruritus|Cholestasis|Cholestatic Liver Disease |
2026-09-01 |
|
| jRCT2051220098 | P3 |
Active, not recruiting |
Alagille Syndrome |
2025-07-31 |
|
| jRCT2031220356 | P3 |
Active, not recruiting |
Cholestasis, Intrahepatic |
2025-07-31 |