Chenodiol
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Also known as Acide chenodesoxycholiqueAcido quenodeoxicolicoAnthropodeoxycholic acidAnthropodesoxycholic acidAnthropododesoxycholic acidChendolChendol 125Chendol 250Chenic acidChenixChenocedonChenocolChenodeoxycholateChenodeoxycholic acidChenodesoxycholic acidChenofalkChenossilCholanormCombidol
Summary
Chenodiol (CHEMBL240597) is an approved small molecule (ATC A05AA01) targeting FPR1, GPBAR1, and NR1H4; indicated across 4 conditions including cerebrotendinous xanthomatosis and inborn errors of metabolism.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- ATC class: A05AA01
- Targets: 3 (FPR1, GPBAR1, NR1H4)
- Indications: 4 conditions
- Clinical trials: 13
- Chemistry: 392.6 Da · C24H40O4
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL240597 |
| Name | Chenodiol |
| Type | Small molecule |
| Max phase | 4 |
| FDA approved | yes |
| PubChem CID | 10133 |
| ChEBI | CHEBI:16755 |
| ATC | A05AA01 |
| Molecular formula | C24H40O4 |
| Molecular weight | 392.6 |
| InChIKey | RUDATBOHQWOJDD-BSWAIDMHSA-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,7R,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 dihydroxy-5β-cholanic acid that is (5β)-cholan-24-oic acid substituted by hydroxy groups at positions 3 and 7 respectively.
Other ChEBI roles (chemical / environmental): human metabolite, mouse metabolite.
Also known as: Acide chenodesoxycholique, Acido quenodeoxicolico, Anthropodeoxycholic acid, Anthropodesoxycholic acid, Anthropododesoxycholic acid, Chendol, Chendol 125, Chendol 250, Chenic acid, Chenix, Chenocedon, Chenocol
Patent coverage: 7,547 distinct patent families (24,403 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 |
|---|---|---|---|---|---|
| FPR1 | FPR1 | Antagonist | 4 | 0% | P21462 |
| GPBAR1 | GPBA receptor | Full agonist | 5.4 | 0.6% | Q8TDU6 |
| NR1H4 | Farnesoid X receptor | Agonist | 5.3 | 0.7% | Q96RI1 |
Broader ChEMBL bioactivity targets: 11 (assay-derived). Sample: Tyrosyl-DNA phosphodiesterase 1, Alpha-2A adrenergic receptor, Bile acid receptor, Menin/Histone-lysine N-methyltransferase MLL, Muscarinic acetylcholine receptor M2, Alpha-1A adrenergic receptor, Ileal sodium/bile acid cotransporter, Bile acid receptor/Retinoic acid receptor RXR-alpha, Bile acid receptor, G-protein coupled bile acid receptor 1.
Bioactivity
ChEMBL activities: 22 potent at pChembl ≥ 5 of 50 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| ADRA2A | 5.52 | AC50 | 3000 | nM | CHEMBL_ACT_25220473 |
| SLC10A2 | 5.48 | Ki | 3300 | nM | CHEMBL_ACT_11002859 |
| NR1H4 | 5.47 | EC50 | 3400 | nM | CHEMBL_ACT_560987 |
| NR1H4 | 5.46 | EC50 | 3500 | nM | CHEMBL_ACT_1494127 |
| GPBAR1 | 5.35 | EC50 | 4430 | nM | CHEMBL_ACT_25994696 |
| GPBAR1 | 5.35 | EC50 | 4430 | nM | CHEMBL_ACT_29078711 |
| GPBAR1 | 5.21 | EC50 | 6100 | nM | CHEMBL_ACT_15165318 |
| NR1H4 | 5.2 | EC50 | 6310 | nM | CHEMBL_ACT_2466793 |
| GPBAR1 | 5.17 | EC50 | 6700 | nM | CHEMBL_ACT_10975102 |
| GPBAR1 | 5.17 | EC50 | 6710 | nM | CHEMBL_ACT_1918822 |
| GPBAR1 | 5.17 | EC50 | 6710 | nM | CHEMBL_ACT_2117826 |
| NR1H4 | 5.15 | EC50 | 7010 | nM | CHEMBL_ACT_18729998 |
| NR1H4 | 5.1 | EC50 | 8000 | nM | CHEMBL_ACT_19129288 |
| NR1H4 | 5.06 | EC50 | 8660 | nM | CHEMBL_ACT_1467362 |
| NR1H4 | 5.06 | EC50 | 8660 | nM | CHEMBL_ACT_1740202 |
| NR1H4 | 5.06 | EC50 | 8660 | nM | CHEMBL_ACT_18373634 |
| NR1H4 | 5.06 | EC50 | 8660 | nM | CHEMBL_ACT_19005349 |
| NR1H4 | 5.06 | EC50 | 8660 | nM | CHEMBL_ACT_454604 |
| NR1H4 | 5.06 | EC50 | 8660 | nM | CHEMBL_ACT_89273 |
| NR1H4 | 5.05 | EC50 | 9000 | nM | CHEMBL_ACT_18067144 |
| NR1H4 | 5 | EC50 | 10000 | nM | CHEMBL_ACT_22450434 |
| NR1H4 | 5 | EC50 | 10000 | nM | CHEMBL_ACT_7943014 |
Target pathways
Aggregated over 3 target gene(s): FPR1, GPBAR1, NR1H4.
Top Reactome pathways
14 total, by targets touching each:
| Pathway | Targets | Genes |
|---|---|---|
| Recycling of bile acids and salts | 1 | NR1H4 |
| Synthesis of bile acids and bile salts | 1 | NR1H4 |
| Synthesis of bile acids and bile salts via 7alpha-hydroxycholesterol | 1 | NR1H4 |
| Synthesis of bile acids and bile salts via 27-hydroxycholesterol | 1 | NR1H4 |
| PPARA activates gene expression | 1 | NR1H4 |
| Endogenous sterols | 1 | NR1H4 |
| Class A/1 (Rhodopsin-like receptors) | 1 | GPBAR1 |
| Nuclear Receptor transcription pathway | 1 | NR1H4 |
| SUMOylation of intracellular receptors | 1 | NR1H4 |
| G alpha (s) signalling events | 1 | GPBAR1 |
| G alpha (i) signalling events | 1 | FPR1 |
| Formyl peptide receptors bind formyl peptides and many other ligands | 1 | FPR1 |
| Interleukin-10 signaling | 1 | FPR1 |
| Neutrophil degranulation | 1 | FPR1 |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| inflammatory response | 2 |
| signal transduction | 2 |
| G protein-coupled receptor signaling pathway | 2 |
| cellular response to bile acid | 2 |
| complement receptor mediated signaling pathway | 1 |
| chemotaxis | 1 |
| adenylate cyclase-modulating G protein-coupled receptor signaling pathway | 1 |
| phospholipase C-activating G protein-coupled receptor signaling pathway | 1 |
| positive regulation of cytosolic calcium ion concentration | 1 |
| obsolete nitric oxide mediated signal transduction | 1 |
| cell communication | 1 |
| signaling | 1 |
| adenylate cyclase-activating G protein-coupled bile acid receptor signaling pathway | 1 |
| positive regulation of ERK1 and ERK2 cascade | 1 |
| energy homeostasis | 1 |
Indications & clinical
Indications
4 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 |
|---|---|---|---|
| cerebrotendinous xanthomatosis | 4 | MONDO:0008948 | MONDO:0008948 |
| inborn errors of metabolism | 4 | MONDO:0019052 | MONDO:0019052 |
| irritable bowel syndrome | 2 | MONDO:0005052 | EFO:0000555 |
| paraplegia | 2 | MONDO:0003757 | HP:0001258 |
Clinical trials
Total trials: 13.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE4 | 4 |
| Not specified | 4 |
| PHASE2 | 2 |
| PHASE3 | 1 |
| EARLY_PHASE1 | 1 |
| PHASE1 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT02340247 | PHASE4 | COMPLETED | Effects of Bile Acids on GLP-1 Secretion After Roux-en-Y Gastric Bypass |
| NCT02876484 | PHASE4 | COMPLETED | Effects of Bile Acids and Bile Acid Sequestrants on GLP-1 Secretion After Roux-en-Y Gastric Bypass |
| NCT02952963 | PHASE4 | COMPLETED | Effect of Bile Acids and Bile Acid Sequstrants on GLP-1 Secretion After Roux-en-Y Gastric Bypass |
| NCT03168555 | PHASE4 | COMPLETED | Changes in Bile Acid Homeostasis and Stool Habits After Cholecystectomy |
| NCT06260748 | PHASE3 | WITHDRAWN | A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX) |
| NCT00004346 | PHASE2 | UNKNOWN | Phase II Study of Cholesterol- and Cholestanol-Free Diet, Lovastatin, and Chenodeoxycholic Acid for Cerebrotendinous Xanthomatosis |
| NCT02314208 | PHASE2 | COMPLETED | Therapeutic Metabolic Intervention in Patients With Spastic Paraplegia SPG5 |
| NCT06180057 | PHASE1 | COMPLETED | Bioequivalence Study to Compare Chenodeoxycholic Acid Capsules (250mg Chenodeoxycholic Acid) Versus Chenodeoxycholic Acid Leadiant 250 mg Hard Capsules (250mg Chenodeoxycholic Acid) |
| NCT00465751 | EARLY_PHASE1 | COMPLETED | Effects of FXR Activation on Hepatic Lipid and Glucose Metabolism |
| NCT00004442 | Not specified | TERMINATED | Study of Bile Acids in Patients With Peroxisomal Disorders |
| NCT00018694 | Not specified | WITHDRAWN | Cholestanol in Humans |
| NCT01666223 | Not specified | COMPLETED | Effect of Bile Acids on GLP-1 Secretion |
| NCT05499026 | Not specified | COMPLETED | Safety and Efficacy of CDCA in CTX Chenodeoxycholic Acid (CDCA) in Patients Affected by Cerebrotendinous Xanthomatosis (CTX) |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.
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.
66 molecules share ≥1 primary target. Top 60 by shared-target count:
| Molecule | Source | Status | Shared targets |
|---|---|---|---|
| Deoxycholic Acid | ChEMBL + PubChem | Phase 4 (approved) | FPR1, GPBAR1, NR1H4 |
| cyclosporine | ChEMBL + PubChem | Phase 4 (approved) | FPR1, NR1H4 |
| CHOLIC ACID | ChEMBL | Phase 4 (approved) | GPBAR1, NR1H4 |
| OBETICHOLIC ACID | ChEMBL | Phase 4 (approved) | GPBAR1, NR1H4 |
| TAURURSODIOL | ChEMBL | Phase 4 (approved) | GPBAR1, NR1H4 |
| TERN-101 | ChEMBL | Phase 2 | GPBAR1, NR1H4 |
| FULVESTRANT | ChEMBL + PubChem | Phase 4 (approved) | NR1H4 |
| REGORAFENIB | ChEMBL + PubChem | Phase 4 (approved) | NR1H4 |
| ACETAMINOPHEN | ChEMBL | Phase 4 (approved) | NR1H4 |
| APREPITANT | ChEMBL | Phase 4 (approved) | FPR1 |
| ATORVASTATIN | ChEMBL | Phase 4 (approved) | NR1H4 |
| BENZBROMARONE | ChEMBL | Phase 4 (approved) | NR1H4 |
| CINACALCET | ChEMBL | Phase 4 (approved) | FPR1 |
| CLOFAZIMINE | ChEMBL | Phase 4 (approved) | NR1H4 |
| CLOTRIMAZOLE | ChEMBL | Phase 4 (approved) | NR1H4 |
| DICLOFENAC | ChEMBL | Phase 4 (approved) | NR1H4 |
| EPALRESTAT | ChEMBL | Phase 4 (approved) | NR1H4 |
| ESTRADIOL | ChEMBL | Phase 4 (approved) | GPBAR1 |
| FELODIPINE | ChEMBL | Phase 4 (approved) | NR1H4 |
| FLUTRIMAZOLE | ChEMBL | Phase 4 (approved) | NR1H4 |
| IVERMECTIN | ChEMBL | Phase 4 (approved) | NR1H4 |
| KETOCONAZOLE | ChEMBL | Phase 4 (approved) | NR1H4 |
| LEVOTHYROXINE | ChEMBL | Phase 4 (approved) | NR1H4 |
| LOPERAMIDE | ChEMBL | Phase 4 (approved) | FPR1 |
| LORATADINE | ChEMBL | Phase 4 (approved) | NR1H4 |
| MONTELUKAST | ChEMBL | Phase 4 (approved) | FPR1 |
| NIMODIPINE | ChEMBL | Phase 4 (approved) | NR1H4 |
| ODEVIXIBAT | ChEMBL | Phase 4 (approved) | NR1H4 |
| PENICILLIN G | ChEMBL | Phase 4 (approved) | FPR1 |
| PERPHENAZINE | ChEMBL | Phase 4 (approved) | FPR1 |
| PHENYLBUTAZONE | ChEMBL | Phase 4 (approved) | FPR1 |
| PRANLUKAST | ChEMBL | Phase 4 (approved) | NR1H4 |
| PRASTERONE | ChEMBL | Phase 4 (approved) | GPBAR1 |
| PREDNISOLONE | ChEMBL | Phase 4 (approved) | GPBAR1 |
| PROGESTERONE | ChEMBL | Phase 4 (approved) | GPBAR1 |
| RALOXIFENE | ChEMBL | Phase 4 (approved) | NR1H4 |
| REPAGLINIDE | ChEMBL | Phase 4 (approved) | NR1H4 |
| RIMONABANT | ChEMBL | Phase 4 (approved) | NR1H4 |
| SIMVASTATIN | ChEMBL | Phase 4 (approved) | NR1H4 |
| SULCONAZOLE | ChEMBL | Phase 4 (approved) | NR1H4 |
| SULFINPYRAZONE | ChEMBL | Phase 4 (approved) | FPR1 |
| SUNITINIB | ChEMBL | Phase 4 (approved) | NR1H4 |
| TESTOSTERONE | ChEMBL | Phase 4 (approved) | GPBAR1 |
| TROGLITAZONE | ChEMBL | Phase 4 (approved) | NR1H4 |
| URSODIOL | ChEMBL | Phase 4 (approved) | GPBAR1 |
| ZAFIRLUKAST | ChEMBL | Phase 4 (approved) | NR1H4 |
| ANDROGRAPHOLIDE | ChEMBL | Phase 3 | NR1H4 |
| CILOFEXOR | ChEMBL | Phase 3 | NR1H4 |
| ELOBIXIBAT | ChEMBL | Phase 3 | NR1H4 |
| GLYCOCHOLIC ACID | ChEMBL | Phase 3 | GPBAR1 |
| NORUCHOLIC ACID | ChEMBL | Phase 3 | GPBAR1 |
| TAUROLITHOCHOLIC ACID | ChEMBL | Phase 3 | GPBAR1 |
| VIDOFLUDIMUS | ChEMBL | Phase 3 | NR1H4 |
| FORETINIB | ChEMBL | Phase 2 | FPR1 |
| NIDUFEXOR | ChEMBL | Phase 2 | NR1H4 |
| NIGULDIPINE | ChEMBL | Phase 2 | FPR1 |
| PX-102 | ChEMBL | Phase 2 | NR1H4 |
| STANOLONE | ChEMBL | Phase 2 | GPBAR1 |
| TAURODEOXYCHOLIC ACID | ChEMBL | Phase 2 | GPBAR1 |
| TROPIFEXOR | ChEMBL | Phase 2 | NR1H4 |
Related Atlas pages
- Genes: FPR1, GPBAR1, NR1H4
- Diseases: cerebrotendinous xanthomatosis, inborn errors of metabolism
- Drugs: Deoxycholic Acid, cyclosporine, Cholic Acid, Obeticholic Acid, Taurursodiol, Fulvestrant, Regorafenib, Acetaminophen, Aprepitant, Atorvastatin, Benzbromarone, Cinacalcet, Clofazimine, Clotrimazole, Diclofenac, Epalrestat, Estradiol, Felodipine, Flutrimazole, Ivermectin, Ketoconazole, Levothyroxine, Loperamide, Loratadine, Montelukast, Nimodipine, Odevixibat, Penicillin G, Perphenazine, Phenylbutazone, Pranlukast, Prasterone, Prednisolone, Progesterone, Raloxifene, Repaglinide, Rimonabant, Simvastatin, Sulconazole, Sulfinpyrazone, Sunitinib, Testosterone, Troglitazone, Ursodiol, Zafirlukast, Andrographolide, Cilofexor, Elobixibat, Norucholic Acid, Taurolithocholic Acid, Vidofludimus