Cholic Acid
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Also known as Cholalic acidCholbamE1000KolbamNSC-6135OrphacolSID26754161SID26754162SID50085904Cholic_acid
Summary
Cholic Acid (CHEMBL205596) is an approved small molecule (ATC A05AA03) targeting GPBAR1 and NR1H4; indicated across 6 conditions including liver disorder and inborn errors of metabolism.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- ATC class: A05AA03
- Targets: 2 (GPBAR1, NR1H4)
- Indications: 6 conditions
- Clinical trials: 6
- Chemistry: 408.6 Da · C24H40O5
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL205596 |
| Name | Cholic Acid |
| Type | Small molecule |
| Max phase | 4 |
| FDA approved | yes |
| PubChem CID | 221493 |
| ChEBI | CHEBI:16359 |
| ATC | A05AA03 |
| Molecular formula | C24H40O5 |
| Molecular weight | 408.6 |
| InChIKey | BHQCQFFYRZLCQQ-OELDTZBJSA-N |
SMILES: C[C@H](CCC(=O)O)[C@H]1CC[C@@H]2[C@@]1([C@H](C[C@H]3[C@H]2[C@@H](C[C@H]4[C@@]3(CC[C@H](C4)O)C)O)O)C
IUPAC name: (4R)-4-[(3R,5S,7R,8R,9S,10S,12S,13R,14S,17R)-3,7,12-trihydroxy-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 that is 5β-cholan-24-oic acid bearing three α-hydroxy substituents at position 3, 7 and 12.
Other ChEBI roles (chemical / environmental): human metabolite, mouse metabolite.
Also known as: Cholalic acid, Cholic acid, Cholbam, E1000, Kolbam, NSC-6135, Orphacol, cholic acid, SID26754161, SID26754162, SID50085904, Cholic Acid
Parent form; salt/anhydrous children: CHEMBL1334097, CHEMBL1421383, CHEMBL3187726
Patent coverage: 29,911 distinct patent families (76,195 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 |
|---|---|---|---|---|---|
| GPBAR1 | GPBA receptor | Full agonist | 5 | 0.6% | Q8TDU6 |
| NR1H4 | Farnesoid X receptor | Agonist | 5 | 0.7% | Q96RI1 |
Broader ChEMBL bioactivity targets: 6 (assay-derived). Sample: G-protein coupled bile acid receptor 1, Bile acid receptor, cGMP-inhibited 3’,5’-cyclic phosphodiesterase 3A, Cytochrome P450 3A4, Cruzipain, G-protein coupled bile acid receptor 1.
Bioactivity
ChEMBL activities: 4 potent at pChembl ≥ 5 of 17 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| Q80SS6 | 5.28 | IC50 | 5300 | nM | CHEMBL_ACT_3500482 |
| GPBAR1 | 5.22 | IC50 | 6000 | nM | CHEMBL_ACT_3500451 |
| GPBAR1 | 5.11 | EC50 | 7720 | nM | CHEMBL_ACT_25994695 |
| GPBAR1 | 5.11 | EC50 | 7720 | nM | CHEMBL_ACT_29078712 |
Target pathways
Aggregated over 2 target gene(s): GPBAR1, NR1H4.
Top Reactome pathways
10 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 |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| cellular response to bile acid | 2 |
| adenylate cyclase-activating G protein-coupled bile acid receptor signaling pathway | 1 |
| positive regulation of ERK1 and ERK2 cascade | 1 |
| energy homeostasis | 1 |
| positive regulation of cholangiocyte proliferation | 1 |
| regulation of bicellular tight junction assembly | 1 |
| signal transduction | 1 |
| G protein-coupled receptor signaling pathway | 1 |
| negative regulation of transcription by RNA polymerase II | 1 |
| intracellular glucose homeostasis | 1 |
| regulation of DNA-templated transcription | 1 |
| regulation of transcription by RNA polymerase II | 1 |
| transcription by RNA polymerase II | 1 |
| inflammatory response | 1 |
| cell-cell junction assembly | 1 |
Indications & clinical
Indications
6 indications (4 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 |
|---|---|---|---|
| liver disorder | 4 | MONDO:0005154 | EFO:0001421 |
| inborn errors of metabolism | 4 | MONDO:0019052 | MONDO:0019052 |
| fatty liver disease | 2 | MONDO:0004790 | HP:0001397 |
| Smith-Lemli-Opitz syndrome | 1 | MONDO:0010035 | MONDO:0010035 |
2 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.
Clinical trials
Total trials: 6.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE3 | 2 |
| Not specified | 2 |
| PHASE2 | 1 |
| PHASE1/PHASE2 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT01115582 | PHASE3 | COMPLETED | Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis |
| NCT01438411 | PHASE3 | COMPLETED | Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis |
| NCT00457639 | PHASE2 | COMPLETED | Cholic Acid for Hepatic Steatosis in Lipodystrophy |
| NCT03720990 | PHASE1/PHASE2 | COMPLETED | Smith-Lemli-Opitz Syndrome and Cholic Acid |
| NCT03115086 | Not specified | ACTIVE_NOT_RECRUITING | The REPLACE Registry for Cholbam® (Cholic Acid) |
| NCT00004442 | Not specified | TERMINATED | Study of Bile Acids in Patients With Peroxisomal Disorders |
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.
55 molecules share ≥1 primary target. Top 55 by shared-target count:
| Molecule | Source | Status | Shared targets |
|---|---|---|---|
| CHENODIOL | ChEMBL + PubChem | Phase 4 (approved) | GPBAR1, NR1H4 |
| DEOXYCHOLIC 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 |
| ATORVASTATIN | ChEMBL | Phase 4 (approved) | NR1H4 |
| BENZBROMARONE | ChEMBL | Phase 4 (approved) | NR1H4 |
| CLOFAZIMINE | ChEMBL | Phase 4 (approved) | NR1H4 |
| CLOTRIMAZOLE | ChEMBL | Phase 4 (approved) | NR1H4 |
| CYCLOSPORINE | 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 |
| LORATADINE | ChEMBL | Phase 4 (approved) | NR1H4 |
| NIMODIPINE | ChEMBL | Phase 4 (approved) | NR1H4 |
| ODEVIXIBAT | ChEMBL | Phase 4 (approved) | NR1H4 |
| 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 |
| 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 |
| NIDUFEXOR | ChEMBL | Phase 2 | NR1H4 |
| PX-102 | ChEMBL | Phase 2 | NR1H4 |
| STANOLONE | ChEMBL | Phase 2 | GPBAR1 |
| TAURODEOXYCHOLIC ACID | ChEMBL | Phase 2 | GPBAR1 |
| TROPIFEXOR | ChEMBL | Phase 2 | NR1H4 |
| TUROFEXORATE ISOPROPYL | ChEMBL | Phase 2 | NR1H4 |
| URSOLIC ACID | ChEMBL | Phase 2 | GPBAR1 |
| Bosentan | PubChem | Approved | NR1H4 |
| Olanzapine | PubChem | Approved | NR1H4 |
| rifampin | PubChem | Approved | NR1H4 |
Related Atlas pages
- Genes: GPBAR1, NR1H4
- Diseases: liver disorder, inborn errors of metabolism
- Drugs: Chenodiol, Deoxycholic Acid, Obeticholic Acid, Taurursodiol, Fulvestrant, Regorafenib, Acetaminophen, Atorvastatin, Benzbromarone, Clofazimine, Clotrimazole, Cyclosporine, Diclofenac, Epalrestat, Estradiol, Felodipine, Flutrimazole, Ivermectin, Ketoconazole, Levothyroxine, Loratadine, Nimodipine, Odevixibat, Pranlukast, Prasterone, Prednisolone, Progesterone, Raloxifene, Repaglinide, Rimonabant, Simvastatin, Sulconazole, Sunitinib, Testosterone, Troglitazone, Ursodiol, Zafirlukast, Andrographolide, Cilofexor, Elobixibat, Norucholic Acid, Taurolithocholic Acid, Vidofludimus, Bosentan, Olanzapine, rifampin