Ursodiol
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Also known as Acide ursodeoxycholiqueAcido ursodeoxicolicoActigallCholursoDestolitNSC-683769UrdoxUrsoUrso 250Urso forteUrsodeoxycholateUrsodeoxycholic acidUrsodesoxycholic acidUrsofalkUrsogalUrsonorment-chenodeoxycholic acid3alpha,7beta-dihydroxycholanic acidSID11532904
Summary
Ursodiol (CHEMBL1551) is an approved small molecule (ATC A05AA02) targeting SLC10A1; indicated across 29 conditions including primary biliary cholangitis and liver disorder.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- ATC class: A05AA02
- Targets: 1 (SLC10A1)
- Indications: 29 conditions
- Clinical trials: 90
- Chemistry: 392.6 Da · C24H40O4
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL1551 |
| Name | Ursodiol |
| Type | Small molecule |
| Max phase | 4 |
| FDA approved | yes |
| PubChem CID | 31401 |
| ChEBI | CHEBI:9907 |
| ATC | A05AA02 |
| Molecular formula | C24H40O4 |
| Molecular weight | 392.6 |
| InChIKey | RUDATBOHQWOJDD-UZVSRGJWSA-N |
SMILES: C[C@H](CCC(=O)O)[C@H]1CC[C@@H]2[C@@]1(CC[C@H]3[C@H]2[C@H](C[C@H]4[C@@]3(CC[C@H](C4)O)C)O)C
IUPAC name: (4R)-4-[(3R,5S,7S,8R,9S,10S,13R,14S,17R)-3,7-dihydroxy-10,13-dimethyl-2,3,4,5,6,7,8,9,11,12,14,15,16,17-tetradecahydro-1H-cyclopenta[a]phenanthren-17-yl]pentanoic acid
ChEBI definition: A bile acid found in the bile of bears (Ursidae) as a conjugate with taurine. Used therapeutically, it prevents the synthesis and absorption of cholesterol and can lead to the dissolution of gallstones.
Other ChEBI roles (chemical / environmental): human metabolite, mouse metabolite.
Also known as: Acide ursodeoxycholique, Acido ursodeoxicolico, Actigall, Cholurso, Destolit, NSC-683769, Urdox, Urso, Urso 250, Urso forte, Ursodeoxycholate, Ursodeoxycholic acid
Parent form; salt/anhydrous children: CHEMBL2028234
Patent coverage: 7,343 distinct patent families (22,553 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.
Targets
Targets
Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).
| Gene | Target | Action | pAffinity | Cancer dependency | UniProt |
|---|---|---|---|---|---|
| SLC10A1 | Sodium/bile acid and sulphated solute cotransporter 1 | Inhibition | 5.44 | 0.1% | Q14973 |
Broader ChEMBL bioactivity targets: 7 (assay-derived). Sample: CDGSH iron-sulfur domain-containing protein 1, Ileal sodium/bile acid cotransporter, Autotaxin, Aminopeptidase N, Hepatic sodium/bile acid cotransporter, G-protein coupled bile acid receptor 1, Bile salt export pump.
Bioactivity
ChEMBL activities: 6 potent at pChembl ≥ 5 of 10 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| CISD1 | 5.95 | Ki | 1134 | nM | CHEMBL_ACT_16766247 |
| GPBAR1 | 5.62 | EC50 | 2390 | nM | CHEMBL_ACT_15165319 |
| O96935 | 5.58 | IC50 | 2600 | nM | CHEMBL_ACT_5030499 |
| ENPP2 | 5.55 | IC50 | 2800 | nM | CHEMBL_ACT_29118998 |
| SLC10A1 | 5.44 | IC50 | 3600 | nM | CHEMBL_ACT_11001416 |
| ENPP2 | 5.03 | IC50 | 9260 | nM | CHEMBL_ACT_25958215 |
Target pathways
Aggregated over 1 target gene(s): SLC10A1.
Top Reactome pathways
1 total, by targets touching each:
| Pathway | Targets | Genes |
|---|---|---|
| Recycling of bile acids and salts | 1 | SLC10A1 |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| bile acid and bile salt transport | 1 |
| response to nutrient levels | 1 |
| response to estrogen | 1 |
| response to ethanol | 1 |
| cellular response to xenobiotic stimulus | 1 |
| monoatomic ion transport | 1 |
| sodium ion transport | 1 |
| lipid transport | 1 |
| symbiont entry into host cell | 1 |
| transmembrane transport | 1 |
Indications & clinical
Indications
29 indications (2 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).
| Indication | Trial phase | MONDO | EFO |
|---|---|---|---|
| primary biliary cholangitis | 4 | MONDO:0005388 | EFO:1001486 |
| liver disorder | 3 | MONDO:0005154 | EFO:0001421 |
| chronic hepatitis C virus infection | 3 | MONDO:0005354 | EFO:0004220 |
| cholelithiasis | 3 | MONDO:0012672 | EFO:0004799 |
| intrahepatic cholestasis | 3 | MONDO:0019072 | MONDO:0019072 |
| type 2 diabetes mellitus | 3 | MONDO:0005148 | MONDO:0005148 |
| gestational diabetes | 3 | MONDO:0005406 | EFO:0004593 |
| sclerosing cholangitis | 2 | MONDO:0018646 | EFO:0004268 |
| congestive heart failure | 2 | MONDO:0005009 | EFO:0000373 |
| metabolic dysfunction-associated steatotic liver disease | 2 | MONDO:0013209 | EFO:0003095 |
| obstructive jaundice | 2 | MONDO:0006874 | EFO:1001068 |
| cholestasis | 2 | MONDO:0001751 | MONDO:0001751 |
| drug-induced liver injury | 2 | MONDO:0005359 | EFO:0004228 |
| rheumatoid arthritis | 2 | MONDO:0008383 | EFO:0000685 |
| diabetes mellitus | 2 | MONDO:0005015 | EFO:0000400 |
| hepatitis D virus infection | 2 | MONDO:0005789 | EFO:0007304 |
| Parkinson disease | 2 | MONDO:0005180 | MONDO:0005180 |
| colorectal neoplasm | 2 | MONDO:0005335 | MONDO:0005575 |
| obesity disorder | 1 | MONDO:0011122 | EFO:0001073 |
| cirrhosis of liver | 1 | MONDO:0005155 | EFO:0001422 |
| glucose intolerance | 1 | MONDO:0001076 | HP:0001952 |
| Huntington disease | 1 | MONDO:0007739 | MONDO:0007739 |
| severe acute respiratory syndrome | 1 | MONDO:0005091 | MONDO:0100096 |
6 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.
Clinical trials
Total trials: 90.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE4 | 19 |
| PHASE2 | 19 |
| Not specified | 19 |
| PHASE1 | 13 |
| PHASE3 | 11 |
| PHASE2/PHASE3 | 7 |
| PHASE1/PHASE2 | 1 |
| EARLY_PHASE1 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT04407650 | PHASE4 | ACTIVE_NOT_RECRUITING | Ursodeoxycholic Acid vs Metformin in Gestational Diabetes Mellitus |
| NCT04650243 | PHASE4 | RECRUITING | Clinical Research of Tapering UDCA in PBC Patients With a Complete Response |
| NCT06517862 | PHASE4 | RECRUITING | Efficacy & Safety of Oral Adjuvants to Phototherapy in Neonatal Hyperbilirubinemia |
| NCT06610760 | PHASE4 | RECRUITING | Efficacy and Safety of Ursodeoxycholic Acid in Reversing Gastric Intestinal Metaplasia |
| NCT00042549 | PHASE4 | TERMINATED | Lithotripsy for the Treatment of Gallstones |
| NCT00161083 | PHASE4 | COMPLETED | UDCA for Symptomatic Gallstone Disease |
| NCT01141322 | PHASE4 | UNKNOWN | Cholestatic Drug-induced Liver Injury |
| NCT01974336 | PHASE4 | COMPLETED | The Preventative and Therapeutical Effect of Ursodeoxycholic Acid(UDCA) to Short Bowel Syndrome Patients |
| NCT02338635 | PHASE4 | UNKNOWN | Effect of Ursodeoxycholic Acid on Peritoneal Function in Patients on Peritoneal Dialysis |
| NCT02340247 | PHASE4 | COMPLETED | Effects of Bile Acids on GLP-1 Secretion After Roux-en-Y Gastric Bypass |
| NCT02415777 | PHASE4 | COMPLETED | Clinical Trial to Evaluate the Efficacy and Safety of ‘URSA SOFT CAP. (UDCA-003)’ in Fatigue Patients With Liver Dysfunction |
| NCT02418130 | PHASE4 | COMPLETED | A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Efficacy and Safety of ‘Ursa Complex Soft Cap. (UDCA-004)’ in Patients With Physical Fatigue. |
| NCT02472509 | PHASE4 | TERMINATED | The Role of Ursodeoxycholic Acid in Treatment of Gallstones in Hemolytic Disorders |
| NCT02748616 | PHASE4 | COMPLETED | C. Difficile and Ursodiol |
| NCT04617561 | PHASE4 | UNKNOWN | Ursodeoxycholic Acid Combined With Low Dose Glucocorticoid in the Treatment of PBC With AIH Features II |
| NCT04786795 | PHASE4 | UNKNOWN | MITE in the Treatment of Dyspepsia After Cholecystectomy |
| NCT04910178 | PHASE4 | COMPLETED | Follow-up of NAFLD Patients With MRI-PDFF |
| NCT04977661 | PHASE4 | COMPLETED | Comparing the Effects of Vitamin E, Ursodeoxycholic Acid and Pentoxyfylline on Egyptian Non-alcoholic Steatohepatitis (NASH) Patients |
| NCT05690646 | PHASE4 | UNKNOWN | Prophylactic Effect of Nirmatrelvir/Ritonavir and Ursodeoxycholic Acid on Reducing Complications After Cardiac Surgery |
| NCT04924868 | PHASE3 | RECRUITING | Ursodeoxycolic Acid for the Prevention of Relapsing Complications After Gallstone Acute Pancreatitis |
| NCT00004748 | PHASE3 | COMPLETED | Low-Dose Oral Methotrexate Versus Colchicine for Primary Biliary Cirrhosis |
| NCT00004784 | PHASE3 | COMPLETED | Phase III Randomized Study of Ursodiol With Vs Without Methotrexate for Primary Biliary Cirrhosis |
| NCT00059202 | PHASE2/PHASE3 | TERMINATED | Trial of High-dose Urso in Primary Sclerosing Cholangitis |
| NCT00134758 | PHASE2/PHASE3 | UNKNOWN | Ursodeoxycholic Acid in the Treatment of Duodenal Adenomas in Familial Adenomatous Polyposis (FAP) Patients |
| NCT00706381 | PHASE3 | COMPLETED | Thyroid Hormones Homeostasis and Energy Metabolism Changes During Stimulation of Endogenously Secreted Bile Acids (BAs) |
| NCT00808743 | PHASE2/PHASE3 | COMPLETED | Prevention of Progression of Duodenal Adenomas in Patients With Familial Adenomatous Polyposis |
| NCT00846963 | PHASE2/PHASE3 | COMPLETED | Ursodiol for Treating Parenteral Nutrition Associated Cholestasis in Neonates |
| NCT01226823 | PHASE3 | WITHDRAWN | Ursodeoxycholic Acid And Cholestasis Of Pregnancy |
| NCT01576458 | PHASE3 | COMPLETED | Ursodeoxycholic Acid in the Treatment of Intrahepatic Cholestasis of Pregnancy |
| NCT01829698 | PHASE3 | COMPLETED | Efficacy and Safety Study of TUDCA Compare UDCA to Treatment Chronic Cholestatic Liver Disease-PBC |
| NCT01857284 | PHASE3 | COMPLETED | Safety and Efficacy of Tauroursodeoxycholic Acid Versus Ursofalk in the Treatment of Adult Primary Biliary Cirrhosis |
| NCT02721862 | PHASE3 | UNKNOWN | Effectiveness of Ursodeoxycholic Acid Use in the Prevention of Gallstone Formation After Sleeve Gastrectomy |
| NCT02937012 | PHASE3 | UNKNOWN | Use of Bezafibrate in Patients With Primary Biliary Cirrhosis to Archive Complete Biochemical Response in Non-responders |
| NCT03056274 | PHASE2/PHASE3 | UNKNOWN | Metformin in Intrahepatic Cholestasis of Pregnancy |
| NCT04542473 | PHASE2/PHASE3 | UNKNOWN | Pancreatic Enzymes and Bile Acids in Acutely Ill Severely Malnourished Children |
| NCT04751188 | PHASE3 | UNKNOWN | A Study to Assess Efficacy and Safety of Bezafibrate in Patients With Primary Biliary Cholangitis |
| NCT05902468 | PHASE2/PHASE3 | UNKNOWN | Ursodeoxycholic Acid as add-on Therapy in Type 2 Diabetes Mellitus |
| NCT00004315 | PHASE2 | UNKNOWN | Phase II Pilot Study to Compare the Bioavailability of Buffered, Enteric-Coated Ursodiol With Unmodified Ursodiol for Chronic Cholestatic Liver Disease and Cystic Fibrosis-Associated Liver Disease |
| NCT00062023 | PHASE2 | TERMINATED | Comparison of Sulindac, Aspirin, and Ursodiol in Preventing Colorectal Cancer |
| NCT00285597 | PHASE2 | UNKNOWN | Ursodeoxycholic Acid in Chronic Heart Failure |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
No CPIC/DPWG dosing guideline, but PharmGKB curates 0 clinical and 2 variant annotation(s) for this drug (gene-keyed; see PharmGKB).
Related molecules
Related molecules
Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.
23 molecules share ≥1 primary target. Top 23 by shared-target count:
| Molecule | Source | Status | Shared targets |
|---|---|---|---|
| CYCLOSPORINE | ChEMBL + PubChem | Phase 4 (approved) | SLC10A1 |
| EZETIMIBE | ChEMBL + PubChem | Phase 4 (approved) | SLC10A1 |
| FUROSEMIDE | ChEMBL + PubChem | Phase 4 (approved) | SLC10A1 |
| IRBESARTAN | ChEMBL + PubChem | Phase 4 (approved) | SLC10A1 |
| PROPRANOLOL | ChEMBL + PubChem | Phase 4 (approved) | SLC10A1 |
| RITONAVIR | ChEMBL + PubChem | Phase 4 (approved) | SLC10A1 |
| ROSIGLITAZONE | ChEMBL + PubChem | Phase 4 (approved) | SLC10A1 |
| ZAFIRLUKAST | ChEMBL + PubChem | Phase 4 (approved) | SLC10A1 |
| FLUVASTATIN | ChEMBL | Phase 4 (approved) | SLC10A1 |
| SULFASALAZINE | ChEMBL | Phase 4 (approved) | SLC10A1 |
| CURCUMIN | ChEMBL + PubChem | Phase 3 (approved) | SLC10A1 |
| FASIGLIFAM | ChEMBL | Phase 3 | SLC10A1 |
| TIRATRICOL | ChEMBL | Phase 3 | SLC10A1 |
| LEVOPROPRANOLOL | ChEMBL | Phase 2 | SLC10A1 |
| Adefovir Dipivoxil | PubChem | Approved | SLC10A1 |
| Bosentan | PubChem | Approved | SLC10A1 |
| Bumetanide | PubChem | Approved | SLC10A1 |
| Caspofungin | PubChem | Approved | SLC10A1 |
| chenodiol | PubChem | Approved | SLC10A1 |
| Deoxycholic Acid | PubChem | Approved | SLC10A1 |
| Macitentan | PubChem | Approved | SLC10A1 |
| Olmesartan | PubChem | Approved | SLC10A1 |
| Progesterone | PubChem | Approved | SLC10A1 |
Related Atlas pages
- Genes: SLC10A1
- Diseases: primary biliary cholangitis, liver disorder, chronic hepatitis C virus infection, cholelithiasis, intrahepatic cholestasis, type 2 diabetes mellitus, gestational diabetes
- Drugs: Cyclosporine, Ezetimibe, Furosemide, Irbesartan, Propranolol, Ritonavir, Rosiglitazone, Zafirlukast, Fluvastatin, Sulfasalazine, Curcumin, Fasiglifam, Tiratricol, Adefovir Dipivoxil, Bosentan, Bumetanide, Caspofungin, chenodiol, Deoxycholic Acid, Macitentan, Olmesartan, Progesterone