Small molecule · Agonist of farnesoid X receptor (NR1H4) · developed by Mirum
Cholic acid is a primary bile acid that acts as an endogenous ligand of the nuclear receptor farnesoid X receptor (FXR), which regulates enzymes and transporters involved in bile acid synthesis and enterohepatic circulation. It supports fat digestion and absorption and enhances bile flow. It is studied in bile acid synthesis disorders, peroxisomal disorders including Zellweger spectrum disorders, and cholestasis.
Summary written with AI from the sources listed at the end of this page.
From the indications section of the FDA label (version of Jan 26, 2026). Read the full label for the exact wording, limitations and safety information.
| Trial | Phase | Condition | Status | Sponsor |
|---|---|---|---|---|
| NCT01438411 | Phase 3 | Bile Acid Synthesis Defect | Completed | MIRM |
| NCT00007020 | Phase 3 | Infantile Refsum's Disease | Completed | MIRM |
| NCT03115086 | N/A | Bile Acid Synthesis Disorders | Active not recruiting | MIRM |
Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.
Bile Acid Synthesis DisordersAdrenoleukodystrophyBile Acid Synthesis DefectCholestasisInfantile Refsum's DiseasePeroxisomal DisordersZellweger Syndrome
Updated Oct 11, 2026. Something wrong or missing? Report it on the MIRM page.
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