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Obeticholic Acid

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Also known as Acide obeticholiqueAcido obeticolicoDSP-1747INT-747Ocaliva6alpha-ethyl-chenodeoxycholic acid6alpha-chenodeoxycholic acid6alpha-ethylchenodeoxycholic acidOBETICHOLIC_acidobeticholic acid (INT-747)

Summary

Obeticholic Acid (CHEMBL566315) is an approved small-molecule farnesoid X receptor agonist (ATC A05AA04) targeting NR1H4; indicated across 14 conditions including primary biliary cholangitis and cholangitis.

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • ATC class: A05AA04
  • Targets: 1 (NR1H4)
  • Indications: 14 conditions
  • Clinical trials: 26
  • Chemistry: 420.6 Da · C26H44O4

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL566315
NameObeticholic Acid
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID447715
ChEBICHEBI:43602
ATCA05AA04
Molecular formulaC26H44O4
Molecular weight420.6
InChIKeyZXERDUOLZKYMJM-ZWECCWDJSA-N

SMILES: CC[C@@H]1[C@@H]2C[C@@H](CC[C@@]2([C@H]3CC[C@]4([C@H]([C@@H]3[C@@H]1O)CC[C@@H]4[C@H](C)CCC(=O)O)C)C)O

IUPAC name: (4R)-4-[(3R,5S,6R,7R,8S,9S,10S,13R,14S,17R)-6-ethyl-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 chenodeoxycholic acid carrying an additional ethyl substituent at the 6α-position. A semi-synthetic bile acid which acts as a farnesoid X receptor agonist and is used for treatment of primary biliary cholangitis.

Pharmacological roles (ChEBI): farnesoid X receptor agonist, hepatoprotective agent.

Also known as: Acide obeticholique, Acido obeticolico, DSP-1747, INT-747, Obeticholic acid, Ocaliva, 6alpha-ethyl-chenodeoxycholic acid, 6alpha-chenodeoxycholic acid, 6alpha-ethylchenodeoxycholic acid, 6alpha-Ethyl-chenodeoxycholic Acid, OBETICHOLIC ACID, obeticholic acid

Patent coverage: 1,370 distinct patent families (3,314 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).

GeneTargetActionpAffinityCancer dependencyUniProt
NR1H4Farnesoid X receptorAgonist70.7%Q96RI1

Broader ChEMBL bioactivity targets: 11 (assay-derived). Sample: G-protein coupled bile acid receptor 1, Alpha-2A adrenergic receptor, Bile acid receptor, Muscarinic acetylcholine receptor M2, 5-hydroxytryptamine receptor 2A, Alpha-1A adrenergic receptor, Adenosine receptor A3, Nuclear receptor subfamily 1 group I member 2, Prostaglandin G/H synthase 1, G-protein coupled bile acid receptor 1.

Bioactivity

ChEMBL activities: 48 potent at pChembl ≥ 5 of 57 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
NR1H48EC5010nMCHEMBL_ACT_18147650
GPBAR17.62EC5023.7nMCHEMBL_ACT_22764494
NR1H47.38EC5042nMCHEMBL_ACT_18147678
NR1H47.12EC5075nMCHEMBL_ACT_28583753
NR1H47.07EC5085nMCHEMBL_ACT_15106119
NR1H47.07EC5085nMCHEMBL_ACT_19005351
NR1H47.07EC5085nMCHEMBL_ACT_25110699
NR1H47.05EC5090nMCHEMBL_ACT_1467481
NR1H47.01EC5098nMCHEMBL_ACT_1740192
NR1H47EC5099nMCHEMBL_ACT_100279
GPBAR17EC50100nMCHEMBL_ACT_13861668
NR1H47EC5099nMCHEMBL_ACT_1494137
NR1H47EC50100nMCHEMBL_ACT_18067145
NR1H47EC5099nMCHEMBL_ACT_19005350
NR1H47EC5099nMCHEMBL_ACT_25110698
NR1H47EC5099nMCHEMBL_ACT_25500477
DGAT27IC5099nMCHEMBL_ACT_29118981
NR1H47EC5099nMCHEMBL_ACT_29165392
NR1H47EC5099nMCHEMBL_ACT_2948252
NR1H47EC5099nMCHEMBL_ACT_458665
NR1H46.82EC50150nMCHEMBL_ACT_22450436
NR1H46.82EC50150nMCHEMBL_ACT_25473322
NR1H46.82EC50152nMCHEMBL_ACT_29190741
NR1H46.82EC50152nMCHEMBL_ACT_29190968
NR1H46.8EC50160nMCHEMBL_ACT_15105990
NR1H46.8EC50160nMCHEMBL_ACT_15636494
NR1H46.75EC50180nMCHEMBL_ACT_18373576
NR1H46.7EC50200nMCHEMBL_ACT_6175483
NR1H46.62EC50237nMCHEMBL_ACT_29190826
NR1H46.54EC50290nMCHEMBL_ACT_22764378

Target pathways

Aggregated over 1 target gene(s): NR1H4.

Top Reactome pathways

8 total, by targets touching each:

PathwayTargetsGenes
Recycling of bile acids and salts1NR1H4
Synthesis of bile acids and bile salts1NR1H4
Synthesis of bile acids and bile salts via 7alpha-hydroxycholesterol1NR1H4
Synthesis of bile acids and bile salts via 27-hydroxycholesterol1NR1H4
PPARA activates gene expression1NR1H4
Endogenous sterols1NR1H4
Nuclear Receptor transcription pathway1NR1H4
SUMOylation of intracellular receptors1NR1H4

Dominant GO biological processes

GO termTargets
negative regulation of transcription by RNA polymerase II1
intracellular glucose homeostasis1
regulation of DNA-templated transcription1
regulation of transcription by RNA polymerase II1
transcription by RNA polymerase II1
inflammatory response1
cell-cell junction assembly1
Notch signaling pathway1
bile acid metabolic process1
negative regulation of tumor necrosis factor-mediated signaling pathway1
negative regulation of very-low-density lipoprotein particle remodeling1
regulation of low-density lipoprotein particle clearance1
bile acid and bile salt transport1
cell differentiation1
intracellular receptor signaling pathway1

Indications & clinical

Indications

14 indications (3 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).

IndicationTrial phaseMONDOEFO
primary biliary cholangitis4MONDO:0005388EFO:0004267
cholangitis4MONDO:0004789HP:0030151
metabolic dysfunction-associated steatotic liver disease3MONDO:0013209EFO:0003095
metabolic dysfunction-associated steatohepatitis3MONDO:0007027EFO:1001249
alcoholic hepatitis2MONDO:0001505EFO:1001345
Barrett esophagus2MONDO:0013662EFO:0000280
sclerosing cholangitis2MONDO:0018646EFO:0004268
familial partial lipodystrophy2MONDO:0020088MONDO:0020088
liver disorder1MONDO:0005154EFO:0001421
obesity disorder1MONDO:0011122EFO:0001073
cholestasis1MONDO:0001751MONDO:0001751

3 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 26.

Phase distribution

PhaseTrials
PHASE213
PHASE34
PHASE14
Not specified4
EARLY_PHASE11

Top trials by phase / activity

NCTPhaseStatusTitle
NCT06691412PHASE3NOT_YET_RECRUITINGObeticholic Acid Among Chronic HBV Patients with Hepatic Steatosis : Clinical and Portal Doppler Outcomes
NCT02548351PHASE3TERMINATEDRandomized Global Phase 3 Study to Evaluate the Impact on NASH With Fibrosis of Obeticholic Acid Treatment
NCT03439254PHASE3COMPLETEDStudy Evaluating the Efficacy and Safety of Obeticholic Acid in Subjects With Compensated Cirrhosis Due to Nonalcoholic Steatohepatitis
NCT04956328PHASE3UNKNOWNStudy of Obeticholic Acid（OCA） Combination With Ursodeoxycholic Acid (UDCA) in Patients With Primay Biliary Cirrhosis (PBC)
NCT04939051PHASE2RECRUITINGObeticholic Acid for Prevention in Barrett’s Esophagus
NCT00501592PHASE2COMPLETEDStudy of INT-747 in Patients With Diabetes and Presumed NAFLD
NCT00550862PHASE2TERMINATEDStudy of INT 747 in Combination With URSO in Patients With Primay Biliary Cirrhosis (PBC)
NCT01265498PHASE2COMPLETEDThe Farnesoid X Receptor (FXR) Ligand Obeticholic Acid in NASH Treatment Trial(FLINT)
NCT01585025PHASE2COMPLETEDObeticholic Acid in Bile Acid Diarrhoea
NCT01625026PHASE2COMPLETEDObeticholic Acid in Bariatric and Gallstone Disease
NCT01865812PHASE2COMPLETEDPhase 2 Study on Effects of Obeticholic Acid (OCA) on Lipoprotein Metabolism in Participants With Primary Biliary Cirrhosis
NCT02430077PHASE2COMPLETEDPhase 2 Study of Obeticholic Acid for Lipodystrophy Patients
NCT02633956PHASE2COMPLETEDCombination Obeticholic Acid (OCA) and Statins for Monitoring of Lipids (CONTROL)
NCT04594694PHASE2TERMINATEDStudy of OCA in Combination With BZF Evaluating Efficacy, Safety, and Tolerability in Participants With PBC
NCT05223036PHASE2TERMINATEDTesting Obeticholic Acid for Familial Adenomatous Polyposis
NCT05239468PHASE2COMPLETEDStudy of OCA in Combination With BZF Evaluating Efficacy, Safety and Tolerability in Participants With PBC
NCT07104786PHASE2COMPLETEDComparison of Effectiveness of Obeticholic Acid With Vitamin E in Patients of Non-alcoholic Fatty Liver Disease
NCT01904539PHASE1COMPLETEDHepatic Impairment Trial of Obeticholic Acid
NCT02532335PHASE1UNKNOWNObeticholic Acid in Morbidly Obese Patients and Healthy Volunteers
NCT04053023PHASE1COMPLETEDLinerixibat and Obeticholic Acid Drug Interaction Study in Healthy Subjects
NCT05133830PHASE1COMPLETEDLinerixibat and Obeticholic Acid Drug Interaction Study in Healthy Adult Participants
NCT03253276EARLY_PHASE1COMPLETEDEffect of Obeticholic Acid on Transport of Bile Acids in PBC Examined by 11C-cholyl-sarcosine PET/CT
NCT03836937Not specifiedCOMPLETEDRole of Obeticholic Acid in the Patients of NAFLD With Raised ALT
NCT04076527Not specifiedUNKNOWNProspective, Multicenter Cohort Study on Primary Biliary Cholangitis
NCT05293938Not specifiedWITHDRAWNA Real-World Data Study to Evaluate the Effectiveness of OCA on Hepatic Outcomes in PBC Patients
NCT05740631Not specifiedUNKNOWNThe Effect of Obeticholic Acid in Healthy Volunteers

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

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.

43 molecules share ≥1 primary target. Top 43 by shared-target count:

MoleculeSourceStatusShared targets
CHENODIOLChEMBL + PubChemPhase 4 (approved)NR1H4
FULVESTRANTChEMBL + PubChemPhase 4 (approved)NR1H4
REGORAFENIBChEMBL + PubChemPhase 4 (approved)NR1H4
ACETAMINOPHENChEMBLPhase 4 (approved)NR1H4
ATORVASTATINChEMBLPhase 4 (approved)NR1H4
BENZBROMARONEChEMBLPhase 4 (approved)NR1H4
CHOLIC ACIDChEMBLPhase 4 (approved)NR1H4
CLOFAZIMINEChEMBLPhase 4 (approved)NR1H4
CLOTRIMAZOLEChEMBLPhase 4 (approved)NR1H4
CYCLOSPORINEChEMBLPhase 4 (approved)NR1H4
DEOXYCHOLIC ACIDChEMBLPhase 4 (approved)NR1H4
DICLOFENACChEMBLPhase 4 (approved)NR1H4
EPALRESTATChEMBLPhase 4 (approved)NR1H4
FELODIPINEChEMBLPhase 4 (approved)NR1H4
FLUTRIMAZOLEChEMBLPhase 4 (approved)NR1H4
IVERMECTINChEMBLPhase 4 (approved)NR1H4
KETOCONAZOLEChEMBLPhase 4 (approved)NR1H4
LEVOTHYROXINEChEMBLPhase 4 (approved)NR1H4
LORATADINEChEMBLPhase 4 (approved)NR1H4
NIMODIPINEChEMBLPhase 4 (approved)NR1H4
ODEVIXIBATChEMBLPhase 4 (approved)NR1H4
PRANLUKASTChEMBLPhase 4 (approved)NR1H4
RALOXIFENEChEMBLPhase 4 (approved)NR1H4
REPAGLINIDEChEMBLPhase 4 (approved)NR1H4
RIMONABANTChEMBLPhase 4 (approved)NR1H4
SIMVASTATINChEMBLPhase 4 (approved)NR1H4
SULCONAZOLEChEMBLPhase 4 (approved)NR1H4
SUNITINIBChEMBLPhase 4 (approved)NR1H4
TAURURSODIOLChEMBLPhase 4 (approved)NR1H4
TROGLITAZONEChEMBLPhase 4 (approved)NR1H4
ZAFIRLUKASTChEMBLPhase 4 (approved)NR1H4
ANDROGRAPHOLIDEChEMBLPhase 3NR1H4
CILOFEXORChEMBLPhase 3NR1H4
ELOBIXIBATChEMBLPhase 3NR1H4
VIDOFLUDIMUSChEMBLPhase 3NR1H4
NIDUFEXORChEMBLPhase 2NR1H4
PX-102ChEMBLPhase 2NR1H4
TERN-101ChEMBLPhase 2NR1H4
TROPIFEXORChEMBLPhase 2NR1H4
TUROFEXORATE ISOPROPYLChEMBLPhase 2NR1H4
BosentanPubChemApprovedNR1H4
OlanzapinePubChemApprovedNR1H4
rifampinPubChemApprovedNR1H4

About this page

Generated deterministically by Sugi Atlas from primary biomedical databases via BioBTree. Every record on this page traces to a primary source.

Generated
Atlas version
v1.1.2
BioBTree
v2.0.2

Sources 30

This page pulls from 30 datasets indexed by BioBTree:

chebichembl_activitychembl_moleculechembl_targetcivic_evidenceclingen_dosageclinical_trialsdepmapefoensemblentrezgogoparentgtopdbgtopdb_interactiongtopdb_ligandhgncmeshmondomsigdbpatent_compoundpharmgkbpharmgkb_guidelinepubchempubchem_activityreactomereactomeparentrefseqtranscriptuniprot