Bezafibrate

Summary

Bezafibrate (CHEMBL264374) is an approved small-molecule antilipemic drug (ATC C10AB02) targeting PPARA; indicated across 8 conditions including cardiovascular disorder and primary biliary cholangitis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: C10AB02
• Targets: 1 (PPARA)
• Indications: 8 conditions
• Clinical trials: 18
• Chemistry: 361.8 Da · C19H20ClNO4

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL264374
Name	Bezafibrate
Type	Small molecule
Max phase	3
FDA approved	yes
PubChem CID	39042
ChEBI	CHEBI:47612
ATC	C10AB02
Molecular formula	C19H20ClNO4
Molecular weight	361.8
InChIKey	IIBYAHWJQTYFKB-UHFFFAOYSA-N

SMILES: CC(C)(C(=O)O)OC1=CC=C(C=C1)CCNC(=O)C2=CC=C(C=C2)Cl

IUPAC name: 2-[4-[2-[(4-chlorobenzoyl)amino]ethyl]phenoxy]-2-methylpropanoic acid

ChEBI definition: A monocarboxylic acid amide obtained by the formal condensation of the carboxy group of 4-chlorobenzoic acid with the amino group of 2-[4-(2-aminoethyl)phenoxy]-2-methylpropanoic acid. Benafibrate is used for the treatment of hyperlipidaemia.

Pharmacological roles (ChEBI): antilipemic drug, geroprotector.

Other ChEBI roles (chemical / environmental): xenobiotic, environmental contaminant.

Also known as: Bezafibrate, Bezafibrato, Bezagen xl, Bezalip, Bezalip-mono, Bezatol sr, BM 15.075, BM-15.075, BM-15075, Fibrazate xl, Liparol xl, NSC-758174

Patent coverage: 5,944 distinct patent families (21,822 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	4.3	0.7%	Q07869

Broader ChEMBL bioactivity targets: 11 (assay-derived). Sample: Survival motor neuron protein, 4’-phosphopantetheinyl transferase ffp, Peroxisome proliferator-activated receptor alpha, Peroxisome proliferator-activated receptor gamma, Peroxisome proliferator-activated receptor alpha, Peroxisome proliferator-activated receptor gamma, Peroxisome proliferator-activated receptor, Aldehyde dehydrogenase 1A1, Peroxisome proliferator-activated receptor delta, Fatty acid-binding protein, intestinal, Fatty acid-binding protein, liver.

Bioactivity

ChEMBL activities: 4 potent at pChembl ≥ 5 of 43 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
SMN1	6.3	Potency	501.2	nM	CHEMBL_ACT_3889330
PPARG	5.98	EC50	1040	nM	CHEMBL_ACT_12066469
PPARG	5.52	EC50	3000	nM	CHEMBL_ACT_2085063
PPARA	5.15	EC50	7100	nM	CHEMBL_ACT_16629378

Target pathways

Aggregated over 1 target gene(s): PPARA.

Top Reactome pathways

13 total, by targets touching each:

Pathway	Targets	Genes
BMAL1:CLOCK,NPAS2 activates circadian expression	1	PPARA
PPARA activates gene expression	1	PPARA
Transcriptional activation of mitochondrial biogenesis	1	PPARA
Activation of gene expression by SREBF (SREBP)	1	PPARA
Transcriptional regulation of white adipocyte differentiation	1	PPARA
Nuclear Receptor transcription pathway	1	PPARA
Regulation of lipid metabolism by PPARalpha	1	PPARA
SUMOylation of intracellular receptors	1	PPARA
Cytoprotection by HMOX1	1	PPARA
Heme signaling	1	PPARA
Transcriptional regulation of brown and beige adipocyte differentiation by EBF2	1	PPARA
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	1
response to hypoxia	1
gluconeogenesis	1
fatty acid metabolic process	1
heart development	1
response to nutrient	1
lactation	1
epidermis development	1
cellular response to starvation	1
hormone-mediated signaling pathway	1
gene expression	1
regulation of ketone metabolic process	1
negative regulation of macrophage derived foam cell differentiation	1
negative regulation of cholesterol storage	1
protein ubiquitination	1

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
cardiovascular disorder	4	MONDO:0004995	EFO:0000319

6 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
sclerosing cholangitis	3	MONDO:0018646	EFO:0004268
myelodysplastic syndrome	2	MONDO:0018881	EFO:0000198
type 2 diabetes mellitus	2	MONDO:0005148	MONDO:0005148
inborn mitochondrial metabolism disorder	2	MONDO:0004069	MONDO:0044970
bipolar disorder	2	MONDO:0004985	MONDO:0004985

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

Clinical trials

Total trials: 18.

Phase distribution

Phase	Trials
PHASE2	7
PHASE3	6
Not specified	3
PHASE4	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02291796	PHASE4	COMPLETED	Bezafibrate for Hyperfibrinogenemia in Acute Myocardial Infarction
NCT02984982	PHASE4	COMPLETED	Evaluation of Effect of Alirocumab on Coronary Atheroma Volume in Japanese Patients Hospitalized for Acute Coronary Syndrome With Hypercholesterolemia
NCT00336167	PHASE3	UNKNOWN	Bezafibrate Trial in CPT2 Deficiency
NCT01654731	PHASE3	COMPLETED	Phase 3 Study of Bezafibrate in Combination With Ursodeoxycholic Acid in Primary Biliary Cirrhosis
NCT02548832	PHASE3	COMPLETED	Bezafibrate Plus Berberine in Mixed Dyslipidemia
NCT02701166	PHASE3	UNKNOWN	The Effect of Bezafibrate on Cholestatic Itch
NCT02937012	PHASE3	UNKNOWN	Use of Bezafibrate in Patients With Primary Biliary Cirrhosis to Archive Complete Biochemical Response in Non-responders
NCT04309773	PHASE3	UNKNOWN	Efficacy of 24 Month of Bezafibrate in Primary Sclerosing Cholangitis With Persistent Cholestasis Despite Ursodeoxycholic Acid Therapy
NCT02481245	PHASE2	RECRUITING	BezafibrateTreatment for Bipolar Depression: A Proof of Concept Study
NCT00506298	PHASE2	COMPLETED	Study of CRx-401 on Glucose Levels in Subjects With Type II Diabetes
NCT00983788	PHASE2	COMPLETED	Effect of Bezafibrate on Muscle Metabolism in Patients With Fatty Acid Oxidation Defects
NCT02398201	PHASE2	COMPLETED	A Study of Bezafibrate in Mitochondrial Myopathy
NCT04594694	PHASE2	TERMINATED	Study of OCA in Combination With BZF Evaluating Efficacy, Safety, and Tolerability in Participants With PBC
NCT04997811	PHASE2	UNKNOWN	Repurposed Drugs to Improve Haematological Responses in Myelodysplastic Syndromes
NCT05239468	PHASE2	COMPLETED	Study of OCA in Combination With BZF Evaluating Efficacy, Safety and Tolerability in Participants With PBC
NCT06858332	Not specified	RECRUITING	Lipoprotein(a) Levels in Patients With Atherosclerotic Cardiovascular Diseases in Russia
NCT01165060	Not specified	COMPLETED	The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD)
NCT03649269	Not specified	COMPLETED	PC-300 Tea Effect on Triglyceride Levels

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 4 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.

37 molecules share ≥1 primary target. Top 37 by shared-target count:

Molecule	Source	Status	Shared targets
SELADELPAR	ChEMBL + PubChem	Phase 4 (approved)	PPARA
BERBERINE	ChEMBL	Phase 4 (approved)	PPARA
CIPROFIBRATE	ChEMBL	Phase 4 (approved)	PPARA
CLOFIBRATE	ChEMBL	Phase 4 (approved)	PPARA
CYCLOSPORINE	ChEMBL	Phase 4 (approved)	PPARA
ELAFIBRANOR	ChEMBL	Phase 4 (approved)	PPARA
FENOFIBRATE	ChEMBL	Phase 4 (approved)	PPARA
FENOFIBRIC ACID	ChEMBL	Phase 4 (approved)	PPARA
GEMFIBROZIL	ChEMBL	Phase 4 (approved)	PPARA
PEMAFIBRATE	ChEMBL	Phase 4 (approved)	PPARA
PIOGLITAZONE	ChEMBL	Phase 4 (approved)	PPARA
RACECADOTRIL	ChEMBL	Phase 4 (approved)	PPARA
ROSIGLITAZONE	ChEMBL	Phase 4 (approved)	PPARA
ALEGLITAZAR	ChEMBL	Phase 3	PPARA
GAMOLENIC ACID	ChEMBL	Phase 3	PPARA
ICOSAPENT	ChEMBL	Phase 3	PPARA
IMIGLITAZAR	ChEMBL	Phase 3	PPARA
LANIFIBRANOR	ChEMBL	Phase 3	PPARA
LOBEGLITAZONE	ChEMBL	Phase 3	PPARA
MURAGLITAZAR	ChEMBL	Phase 3	PPARA
TESAGLITAZAR	ChEMBL	Phase 3	PPARA
CLOFIBRIC ACID	ChEMBL	Phase 2	PPARA
DIHOMO-GAMMA-LINOLENIC ACID	ChEMBL	Phase 2	PPARA
FARGLITAZAR	ChEMBL	Phase 2	PPARA
GW501516	ChEMBL	Phase 2	PPARA
GW590735	ChEMBL	Phase 2	PPARA
INDEGLITAZAR	ChEMBL	Phase 2	PPARA
LINOLEIC ACID	ChEMBL	Phase 2	PPARA
LY-518674	ChEMBL	Phase 2	PPARA
NAVEGLITAZAR	ChEMBL	Phase 2	PPARA
OLEIC ACID	ChEMBL	Phase 2	PPARA
PIRINIXIC ACID	ChEMBL	Phase 2	PPARA
RAGAGLITAZAR	ChEMBL	Phase 2	PPARA
REGLITAZAR	ChEMBL	Phase 2	PPARA
URSOLIC ACID	ChEMBL	Phase 2	PPARA
Bosentan	PubChem	Approved	PPARA
regorafenib	PubChem	Approved	PPARA