Elafibranor

Summary

Elafibranor (CHEMBL3707395) is an approved small molecule (ATC A05AX06) targeting PPARA and PPARG; indicated across 7 conditions including cholangitis and primary biliary cholangitis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: A05AX06
• Targets: 2 (PPARA, PPARG)
• Indications: 7 conditions
• Clinical trials: 22
• Chemistry: 384.5 Da · C22H24O4S

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3707395
Name	Elafibranor
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	9864881
ATC	A05AX06
Molecular formula	C22H24O4S
Molecular weight	384.5
InChIKey	AFLFKFHDSCQHOL-IZZDOVSWSA-N

SMILES: CC1=CC(=CC(=C1OC(C)(C)C(=O)O)C)/C=C/C(=O)C2=CC=C(C=C2)SC

IUPAC name: 2-[2,6-dimethyl-4-[(E)-3-(4-methylsulfanylphenyl)-3-oxoprop-1-enyl]phenoxy]-2-methylpropanoic acid

Also known as: Elafibranor, GFT-505, Gft505, GFT505, Iqirvo, ELAFIBRANOR

Patent coverage: 685 distinct patent families (1,948 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
PPARA	Peroxisome proliferator-activated receptor-α	Agonist	8.22	0.7%	Q07869
PPARG	Peroxisome proliferator-activated receptor-γ	Agonist	7.33	2.6%	P37231

Broader ChEMBL bioactivity targets: 4 (assay-derived). Sample: Peroxisome proliferator-activated receptor gamma, Peroxisome proliferator-activated receptor alpha, NACHT, LRR and PYD domains-containing protein 3, Peroxisome proliferator-activated receptor delta.

Bioactivity

ChEMBL activities: 22 potent at pChembl ≥ 5 of 23 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
PPARA	8	EC50	10	nM	CHEMBL_ACT_19005344
PPARD	8	EC50	10	nM	CHEMBL_ACT_19005345
PPARA	7.35	EC50	45	nM	CHEMBL_ACT_25110711
PPARD	6.94	EC50	115	nM	CHEMBL_ACT_18854508
PPARA	6.89	EC50	130	nM	CHEMBL_ACT_26335927
PPARG	6.76	EC50	175	nM	CHEMBL_ACT_25110712
PPARD	6.68	EC50	210	nM	CHEMBL_ACT_26335928
PPARA	6.65	EC50	225	nM	CHEMBL_ACT_18854517
PPARD	6.6	EC50	249	nM	CHEMBL_ACT_23260708
PPARA	6.43	EC50	375	nM	CHEMBL_ACT_23260654
PPARG	6.39	EC50	410	nM	CHEMBL_ACT_26335926
PPARD	6.14	EC50	730	nM	CHEMBL_ACT_23159518
PPARA	6.12	EC50	760	nM	CHEMBL_ACT_23159498
PPARD	6.11	EC50	780.8	nM	CHEMBL_ACT_29278714
PPARA	6.09	EC50	820.3	nM	CHEMBL_ACT_24405047
PPARD	6.07	EC50	842.9	nM	CHEMBL_ACT_24405092
PPARA	6.05	EC50	888.4	nM	CHEMBL_ACT_29278681
PPARG	5.8	EC50	1566	nM	CHEMBL_ACT_29278746
PPARG	5.73	EC50	1844	nM	CHEMBL_ACT_24405120
PPARG	5.67	EC50	2116	nM	CHEMBL_ACT_18854504
PPARG	5.55	EC50	2790	nM	CHEMBL_ACT_23159538
PPARG	5.4	EC50	3962	nM	CHEMBL_ACT_23260678

Target pathways

Aggregated over 2 target gene(s): PPARA, PPARG.

Top Reactome pathways

16 total, by targets touching each:

Pathway	Targets	Genes
PPARA activates gene expression	2	PPARA, PPARG
Transcriptional regulation of white adipocyte differentiation	2	PPARA, PPARG
Nuclear Receptor transcription pathway	2	PPARA, PPARG
SUMOylation of intracellular receptors	2	PPARA, PPARG
Transcriptional regulation of brown and beige adipocyte differentiation by EBF2	2	PPARA, PPARG
BMAL1:CLOCK,NPAS2 activates circadian expression	1	PPARA
Transcriptional activation of mitochondrial biogenesis	1	PPARA
Activation of gene expression by SREBF (SREBP)	1	PPARA
Regulation of lipid metabolism by PPARalpha	1	PPARA
Regulation of PTEN gene transcription	1	PPARG
MECP2 regulates transcription factors	1	PPARG
Cytoprotection by HMOX1	1	PPARA
Heme signaling	1	PPARA
MLL4 and MLL3 complexes regulate expression of PPARG target genes in adipogenesis and hepatic steatosis	1	PPARG
Expression of BMAL (ARNTL), CLOCK, and NPAS2	1	PPARA
RORA,B,C and NR1D1 (REV-ERBA) regulate gene expression	1	PPARA

Dominant GO biological processes

GO term	Targets
negative regulation of transcription by RNA polymerase II	2
fatty acid metabolic process	2
heart development	2
response to nutrient	2
hormone-mediated signaling pathway	2
negative regulation of macrophage derived foam cell differentiation	2
negative regulation of cholesterol storage	2
cell differentiation	2
negative regulation of transforming growth factor beta receptor signaling pathway	2
intracellular receptor signaling pathway	2
peroxisome proliferator activated receptor signaling pathway	2
regulation of circadian rhythm	2
positive regulation of DNA-templated transcription	2
positive regulation of fatty acid metabolic process	2
positive regulation of transcription by RNA polymerase II	2

Indications & clinical

Indications

1 approved indication. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
cholangitis	4	MONDO:0004789	MONDO:0004789

5 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
primary biliary cholangitis	3	MONDO:0005388	EFO:1001486
metabolic dysfunction-associated steatotic liver disease	2	MONDO:0013209	EFO:0003095
type 2 diabetes mellitus	2	MONDO:0005148	MONDO:0005148
sclerosing cholangitis	2	MONDO:0018646	EFO:0004268
liver disorder	1	MONDO:0005154	EFO:0001421

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

Clinical trials

Total trials: 22.

Phase distribution

Phase	Trials
PHASE2	9
PHASE1	7
PHASE3	6

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04526665	PHASE3	ACTIVE_NOT_RECRUITING	Study of Elafibranor in Patients With Primary Biliary Cholangitis (PBC)
NCT06016842	PHASE3	RECRUITING	A Long-Term Study of Elafibranor in Adult Participants With Primary Biliary Cholangitis
NCT06383403	PHASE3	ACTIVE_NOT_RECRUITING	A Study of Elafibranor in Adults With Primary Biliary Cholangitis and Inadequate Response or Intolerance to Ursodeoxycholic Acid.
NCT06730061	PHASE3	ACTIVE_NOT_RECRUITING	A Study of Elafibranor in Adult Japanese Participants With Primary Biliary Cholangitis (PBC)
NCT07387549	PHASE3	RECRUITING	A Study to Assess How Well and Safely Elafibranor Works in Adult Participants With Primary Sclerosing Cholangitis
NCT02704403	PHASE3	TERMINATED	Phase 3 Study to Evaluate the Efficacy and Safety of Elafibranor Versus Placebo in Patients With Nonalcoholic Steatohepatitis (NASH)
NCT05627362	PHASE2	ACTIVE_NOT_RECRUITING	A Study to Assess Safety and Effectiveness of Elafibranor in Adult Participants With Primary Sclerosing Cholangitis.
NCT01261494	PHASE2	COMPLETED	Study With GFT505 (80mg) Versus Placebo in Patients With Type 2 Diabetes Mellitus
NCT01271751	PHASE2	COMPLETED	Pilot Study With GFT505 (80mg) in Atherogenic Dyslipidaemic Patients With Abdominal Obesity
NCT01271777	PHASE2	COMPLETED	Pilot Study With GFT505 (80mg) in Patients With Insulin Resistance and Abdominal Obesity
NCT01275469	PHASE2	COMPLETED	Pilot Study With GFT505 (80mg) in Patients Presenting With Impaired Glucose Tolerance and Abdominal Obesity.
NCT01694849	PHASE2	COMPLETED	Phase IIb Study to Evaluate the Efficacy and Safety of GFT505 Versus Placebo in Patients With Non-Alcoholic Steatohepatitis (NASH)
NCT03124108	PHASE2	COMPLETED	Study to Evaluate the Efficacy and Safety of Elafibranor in Patients With Primary Biliary Cholangitis (PBC) and Inadequate Response to Ursodeoxycholic Acid
NCT03883607	PHASE2	TERMINATED	Elafibranor, PK and Safety in Children and Adolescents 8 to 17 Years of Age With Non Alcoholic Steatohepatitis (NASH)
NCT03953456	PHASE2	TERMINATED	Study to Evaluate the Effect of Elafibranor on Hepatic Lipid Composition in Subjects With Nonalcoholic Fatty Liver (NAFL)
NCT02091310	PHASE1	COMPLETED	Phase I Study to Evaluate the Effect of GFT505 on QT/QTc Interval in Healthy Volunteers
NCT03765671	PHASE1	COMPLETED	Elafibranor Pharmacokinetic Parameters in Hepatic Impaired Patients
NCT03844555	PHASE1	COMPLETED	Elafibranor Pharmacokinetic Parameters in Renal Impaired Patients
NCT03985969	PHASE1	COMPLETED	Study to Investigate the Potential Drug-Drug Interaction Between Elafibranor and Indomethacin
NCT04171752	PHASE1	COMPLETED	Elafibranor Pharmacokinetic Parameters in Elderly Healthy Volunteers
NCT05543369	PHASE1	COMPLETED	Study to Compare the Level of Elafibranor in Blood After Repeat Administration in Japanese and Non-Asian Healthy Participants
NCT05564208	PHASE1	COMPLETED	A Study to Evaluate the Effect of Food on the Level of Circulating Elafibranor in Healthy Participants After Intake of a Single Tablet

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

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.

93 molecules share ≥1 primary target. Top 93 by shared-target count:

Molecule	Source	Status	Shared targets
FENOFIBRATE	ChEMBL	Phase 4 (approved)	PPARA, PPARG
FENOFIBRIC ACID	ChEMBL	Phase 4 (approved)	PPARA, PPARG
GEMFIBROZIL	ChEMBL	Phase 4 (approved)	PPARA, PPARG
PEMAFIBRATE	ChEMBL	Phase 4 (approved)	PPARA, PPARG
PIOGLITAZONE	ChEMBL	Phase 4 (approved)	PPARA, PPARG
ROSIGLITAZONE	ChEMBL	Phase 4 (approved)	PPARA, PPARG
ALEGLITAZAR	ChEMBL	Phase 3	PPARA, PPARG
BEZAFIBRATE	ChEMBL	Phase 3	PPARA, PPARG
GAMOLENIC ACID	ChEMBL	Phase 3	PPARA, PPARG
ICOSAPENT	ChEMBL	Phase 3	PPARA, PPARG
IMIGLITAZAR	ChEMBL	Phase 3	PPARA, PPARG
LANIFIBRANOR	ChEMBL	Phase 3	PPARA, PPARG
LOBEGLITAZONE	ChEMBL	Phase 3	PPARA, PPARG
MURAGLITAZAR	ChEMBL	Phase 3	PPARA, PPARG
TESAGLITAZAR	ChEMBL	Phase 3	PPARA, PPARG
DIHOMO-GAMMA-LINOLENIC ACID	ChEMBL	Phase 2	PPARA, PPARG
FARGLITAZAR	ChEMBL	Phase 2	PPARA, PPARG
GW501516	ChEMBL	Phase 2	PPARA, PPARG
GW590735	ChEMBL	Phase 2	PPARA, PPARG
INDEGLITAZAR	ChEMBL	Phase 2	PPARA, PPARG
LINOLEIC ACID	ChEMBL	Phase 2	PPARA, PPARG
LY-518674	ChEMBL	Phase 2	PPARA, PPARG
NAVEGLITAZAR	ChEMBL	Phase 2	PPARA, PPARG
OLEIC ACID	ChEMBL	Phase 2	PPARA, PPARG
PIRINIXIC ACID	ChEMBL	Phase 2	PPARA, PPARG
RAGAGLITAZAR	ChEMBL	Phase 2	PPARA, PPARG
REGLITAZAR	ChEMBL	Phase 2	PPARA, PPARG
FULVESTRANT	ChEMBL + PubChem	Phase 4 (approved)	PPARG
SELADELPAR	ChEMBL + PubChem	Phase 4 (approved)	PPARA
BENZBROMARONE	ChEMBL	Phase 4 (approved)	PPARG
BERBERINE	ChEMBL	Phase 4 (approved)	PPARA
BEXAROTENE	ChEMBL	Phase 4 (approved)	PPARG
CANDESARTAN CILEXETIL	ChEMBL	Phase 4 (approved)	PPARG
CANNABIDIOL	ChEMBL	Phase 4 (approved)	PPARG
CARVEDILOL	ChEMBL	Phase 4 (approved)	PPARG
CEFAMANDOLE	ChEMBL	Phase 4 (approved)	PPARG
CEFOTAXIME	ChEMBL	Phase 4 (approved)	PPARG
CEFOXITIN	ChEMBL	Phase 4 (approved)	PPARG
CEFTAZIDIME	ChEMBL	Phase 4 (approved)	PPARG
CEFTRIAXONE	ChEMBL	Phase 4 (approved)	PPARG
CIPROFIBRATE	ChEMBL	Phase 4 (approved)	PPARA
CLOBETASOL PROPIONATE	ChEMBL	Phase 4 (approved)	PPARG
CLOFIBRATE	ChEMBL	Phase 4 (approved)	PPARA
CYCLOSPORINE	ChEMBL	Phase 4 (approved)	PPARA
EFAVIRENZ	ChEMBL	Phase 4 (approved)	PPARG
GLYBURIDE	ChEMBL	Phase 4 (approved)	PPARG
INDOMETHACIN	ChEMBL	Phase 4 (approved)	PPARG
LASOFOXIFENE	ChEMBL	Phase 4 (approved)	PPARG
LEVOTHYROXINE	ChEMBL	Phase 4 (approved)	PPARG
LIOTHYRONINE	ChEMBL	Phase 4 (approved)	PPARG
LUMIRACOXIB	ChEMBL	Phase 4 (approved)	PPARG
MASOPROCOL	ChEMBL	Phase 4 (approved)	PPARG
METHYLENE BLUE	ChEMBL	Phase 4 (approved)	PPARG
MONTELUKAST	ChEMBL	Phase 4 (approved)	PPARG
NINTEDANIB	ChEMBL	Phase 4 (approved)	PPARG
RACECADOTRIL	ChEMBL	Phase 4 (approved)	PPARA
RIMONABANT	ChEMBL	Phase 4 (approved)	PPARG
SULINDAC	ChEMBL	Phase 4 (approved)	PPARG
TELMISARTAN	ChEMBL	Phase 4 (approved)	PPARG
TERIFLUNOMIDE	ChEMBL	Phase 4 (approved)	PPARG
TIPRANAVIR	ChEMBL	Phase 4 (approved)	PPARG
TROGLITAZONE	ChEMBL	Phase 4 (approved)	PPARG
ZAFIRLUKAST	ChEMBL	Phase 4 (approved)	PPARG
BALAGLITAZONE	ChEMBL	Phase 3	PPARG
CANDESARTAN	ChEMBL	Phase 3	PPARG
DOCONEXENT	ChEMBL	Phase 3	PPARG
LERIGLITAZONE	ChEMBL	Phase 3	PPARG
NAMODENOSON	ChEMBL	Phase 3	PPARG
QUERCETIN	ChEMBL	Phase 3	PPARG
RESVERATROL	ChEMBL	Phase 3	PPARG
RIVOGLITAZONE	ChEMBL	Phase 3	PPARG
TIRATRICOL	ChEMBL	Phase 3	PPARG
ARHALOFENATE	ChEMBL	Phase 2	PPARG
ATX08-001	ChEMBL	Phase 2	PPARG
CANNABIGEROL	ChEMBL	Phase 2	PPARG
CIGLITAZONE	ChEMBL	Phase 2	PPARG
CLOFIBRIC ACID	ChEMBL	Phase 2	PPARA
CXA-10	ChEMBL	Phase 2	PPARG
EFATUTAZONE	ChEMBL	Phase 2	PPARG
ENGLITAZONE	ChEMBL	Phase 2	PPARG
HALOFENATE	ChEMBL	Phase 2	PPARG
INT131	ChEMBL	Phase 2	PPARG
IRX-4204	ChEMBL	Phase 2	PPARG
MITOGLITAZONE	ChEMBL	Phase 2	PPARG
MK-0533	ChEMBL	Phase 2	PPARG
URSOLIC ACID	ChEMBL	Phase 2	PPARA
Afatinib	PubChem	Approved	PPARG
Apixaban	PubChem	Approved	PPARG
Binimetinib	PubChem	Approved	PPARG
Bosentan	PubChem	Approved	PPARA
chenodiol	PubChem	Approved	PPARG
Imipenem	PubChem	Approved	PPARG
regorafenib	PubChem	Approved	PPARA