Aminoglutethimide

Summary

Aminoglutethimide (CHEMBL488) is an approved small-molecule antineoplastic agent (ATC L02BG01) targeting CYP11A1 and CYP19A1; indicated across 3 conditions including neoplasm and breast neoplasm; with CIViC clinical evidence for 1 variant-indication association (e.g. TFF3 EXPRESSION in breast cancer).

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: L02BG01
• Targets: 2 (CYP11A1, CYP19A1)
• Indications: 3 conditions
• Clinical trials: 2
• Precision-oncology evidence (CIViC): 1 variant–indication association
• Chemistry: 232.28 Da · C13H16N2O2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL488
Name	Aminoglutethimide
Type	Small molecule
Max phase	4
FDA approved	no
PubChem CID	2145
ChEBI	CHEBI:2654
ATC	L02BG01
Molecular formula	C13H16N2O2
Molecular weight	232.28
InChIKey	ROBVIMPUHSLWNV-UHFFFAOYSA-N

SMILES: CCC1(CCC(=O)NC1=O)C2=CC=C(C=C2)N

IUPAC name: 3-(4-aminophenyl)-3-ethylpiperidine-2,6-dione

ChEBI definition: A dicarboximide that is a six-membered cyclic compound having ethyl and 4-aminophenyl substituents at the 3-position.

Pharmacological roles (ChEBI): antineoplastic agent, adrenergic agent, EC 1.14.14.14 (aromatase) inhibitor, anticonvulsant.

Also known as: Aminoglutethimide, Aminoglutetimida, Cytadren, NSC-330915, Orimeten, aminoglutethimide, SID26747446, SID50105852, SID85230926, Amino-glutethimide, AMINOGLUTETHIMIDE, SID90340970

Patent coverage: 18,293 distinct patent families (74,271 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
CYP11A1	CYP11A1	Inhibition	4.52	0.2%	P05108
CYP19A1	CYP19A1	Inhibition		0%	P11511

Broader ChEMBL bioactivity targets: 11 (assay-derived). Sample: Peripheral myelin protein 22, Thromboxane-A synthase, Aromatase, Cholesterol side-chain cleavage enzyme, mitochondrial, cGMP-inhibited 3’,5’-cyclic phosphodiesterase 3A, 3’,5’-cyclic-AMP phosphodiesterase 4D, Cytochrome P450 11B2, mitochondrial, Cytochrome P450 3A4, Steroid 17-alpha-hydroxylase/17,20 lyase, Aromatase.

Bioactivity

ChEMBL activities: 35 potent at pChembl ≥ 5 of 65 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
P22443	6.58	EC50	260	nM	CHEMBL_ACT_719199
CYP19A1	6.57	IC50	270	nM	CHEMBL_ACT_1885050
CYP19A1	6.57	IC50	270	nM	CHEMBL_ACT_2279870
CYP19A1	6.33	Ki	470	nM	CHEMBL_ACT_1099394
CYP19A1	6.22	Ki	600	nM	CHEMBL_ACT_160554
CYP19A1	6.22	Ki	600	nM	CHEMBL_ACT_195131
CYP19A1	6.22	Ki	600	nM	CHEMBL_ACT_595813
CYP19A1	6.17	Ki	680	nM	CHEMBL_ACT_1115197
CYP19A1	5.85	Ki	1410	nM	CHEMBL_ACT_1463459
CYP19A1	5.85	Ki	1410	nM	CHEMBL_ACT_1483240
CYP19A1	5.77	IC50	1700	nM	CHEMBL_ACT_1099392
CYP19A1	5.75	Ki	1800	nM	CHEMBL_ACT_195132
CYP19A1	5.75	Ki	1800	nM	CHEMBL_ACT_895828
CYP19A1	5.72	IC50	1900	nM	CHEMBL_ACT_325803
CYP19A1	5.72	IC50	1900	nM	CHEMBL_ACT_478855
CYP19A1	5.55	IC50	2800	nM	CHEMBL_ACT_1463457
CYP19A1	5.55	IC50	2800	nM	CHEMBL_ACT_1483238
CYP19A1	5.39	Ki	4100	nM	CHEMBL_ACT_16432265
CYP19A1	5.28	IC50	5200	nM	CHEMBL_ACT_1128695
CYP19A1	5.28	IC50	5200	nM	CHEMBL_ACT_290938
CYP19A1	5.24	IC50	5800	nM	CHEMBL_ACT_6219415
CYP19A1	5.21	IC50	6130	nM	CHEMBL_ACT_1115196
P22443	5.21	IC50	6200	nM	CHEMBL_ACT_789248
CYP19A1	5.19	IC50	6400	nM	CHEMBL_ACT_15156701
CYP19A1	5.19	IC50	6400	nM	CHEMBL_ACT_2201703
CYP19A1	5.16	IC50	7000	nM	CHEMBL_ACT_18102349
CYP19A1	5.1	IC50	8000	nM	CHEMBL_ACT_195129
CYP19A1	5.1	IC50	8000	nM	CHEMBL_ACT_895825
CYP19A1	5.08	IC50	8300	nM	CHEMBL_ACT_1115198
CYP19A1	5.05	IC50	9000	nM	CHEMBL_ACT_18102374

Target pathways

Aggregated over 2 target gene(s): CYP11A1, CYP19A1.

Top Reactome pathways

5 total, by targets touching each:

Pathway	Targets	Genes
Endogenous sterols	2	CYP11A1, CYP19A1
Estrogen biosynthesis	1	CYP19A1
Pregnenolone biosynthesis	1	CYP11A1
Defective CYP11A1 causes AICSR	1	CYP11A1
Defective CYP19A1 causes AEXS	1	CYP19A1

Dominant GO biological processes

GO term	Targets
sterol metabolic process	2
lipid metabolic process	2
steroid biosynthetic process	2
C21-steroid hormone biosynthetic process	1
glucocorticoid biosynthetic process	1
cholesterol metabolic process	1
cortisol metabolic process	1
vitamin D metabolic process	1
cellular response to peptide hormone stimulus	1
steroid hormone biosynthetic process	1
alcohol metabolic process	1
steroid metabolic process	1
C21-steroid hormone metabolic process	1
negative regulation of chronic inflammatory response	1
estrogen biosynthetic process	1

Indications & clinical

Indications

3 indications (1 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
neoplasm	4	MONDO:0005070	EFO:0000616
breast neoplasm	3	MONDO:0021100	MONDO:0007254

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

Clinical trials

Total trials: 2.

Phase distribution

Phase	Trials
PHASE3	1
PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00309491	PHASE3	COMPLETED	Randomized Study Comparing Tamoxifen vs. Tamoxifen + Aminoglutethimide in Postmenopausal Receptor-positive Patients
NCT00006371	PHASE2	TERMINATED	A Phase II Trial of Early Medical Adrenalectomy for D0.5 Prostate Cancer

Clinical evidence (CIViC)

Variant × indication × effect (1 predictive associations from 1 curated evidence items):

Variant	Indication	Effect	Therapy	Level	CIViC
TFF3 EXPRESSION	Breast Cancer	Sensitivity/Response	Aminoglutethimide + Tamoxifen	CIViC B	EID823

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.

40 molecules share ≥1 primary target. Top 40 by shared-target count:

Molecule	Source	Status	Shared targets
ANASTROZOLE	ChEMBL	Phase 4 (approved)	CYP19A1
CLOTRIMAZOLE	ChEMBL	Phase 4 (approved)	CYP19A1
ESTRONE	ChEMBL	Phase 4 (approved)	CYP19A1
EXEMESTANE	ChEMBL	Phase 4 (approved)	CYP19A1
FLUCONAZOLE	ChEMBL	Phase 4 (approved)	CYP19A1
FLUOROURACIL	ChEMBL	Phase 4 (approved)	CYP19A1
KETOCONAZOLE	ChEMBL	Phase 4 (approved)	CYP19A1
LETROZOLE	ChEMBL	Phase 4 (approved)	CYP19A1
MICONAZOLE	ChEMBL	Phase 4 (approved)	CYP19A1
NIMESULIDE	ChEMBL	Phase 4 (approved)	CYP19A1
OSILODROSTAT	ChEMBL	Phase 4 (approved)	CYP19A1
POSACONAZOLE	ChEMBL	Phase 4 (approved)	CYP19A1
TESTOLACTONE	ChEMBL	Phase 4 (approved)	CYP19A1
TESTOSTERONE	ChEMBL	Phase 4 (approved)	CYP19A1
DOCONEXENT	ChEMBL	Phase 3	CYP19A1
ENDOXIFEN	ChEMBL	Phase 3	CYP19A1
ICOSAPENT	ChEMBL	Phase 3	CYP19A1
QUERCETIN	ChEMBL	Phase 3	CYP19A1
RESVERATROL	ChEMBL	Phase 3	CYP19A1
2-METHOXYESTRADIOL	ChEMBL	Phase 2	CYP19A1
AZALANSTAT	ChEMBL	Phase 2	CYP19A1
DAIDZEIN	ChEMBL	Phase 2	CYP19A1
DEXFADROSTAT	ChEMBL	Phase 2	CYP19A1
DOCOSAPENTAENOIC ACID	ChEMBL	Phase 2	CYP19A1
FADROZOLE	ChEMBL	Phase 2	CYP19A1
FLAVONE	ChEMBL	Phase 2	CYP19A1
FORMESTANE	ChEMBL	Phase 2	CYP19A1
GENISTEIN	ChEMBL	Phase 2	CYP19A1
IROSUSTAT	ChEMBL	Phase 2	CYP19A1
LIAROZOLE	ChEMBL	Phase 2	CYP19A1
LINOLEIC ACID	ChEMBL	Phase 2	CYP19A1
LUTEOLIN	ChEMBL	Phase 2	CYP19A1
OLEIC ACID	ChEMBL	Phase 2	CYP19A1
PINOCEMBRIN	ChEMBL	Phase 2	CYP19A1
PLOMESTANE	ChEMBL	Phase 2	CYP19A1
ROGLETIMIDE	ChEMBL	Phase 2	CYP19A1
STANOLONE	ChEMBL	Phase 2	CYP19A1
URSOLIC ACID	ChEMBL	Phase 2	CYP19A1
VOROZOLE	ChEMBL	Phase 2	CYP19A1
Fulvestrant	PubChem	Approved	CYP19A1