Cyproterone Acetate

Summary

Cyproterone Acetate (CHEMBL139835) is a phase-3 clinical-stage small-molecule androgen antagonist (ATC G03HA01) targeting AR; indicated across 8 conditions including prostate adenocarcinoma and endometriosis; with CIViC clinical evidence for 1 variant-indication association (e.g. AR Mutation in prostate cancer).

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• ATC class: G03HA01
• Targets: 1 (AR)
• Indications: 8 conditions
• Clinical trials: 29
• Precision-oncology evidence (CIViC): 1 variant–indication association
• Chemistry: 416.9 Da · C24H29ClO4

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL139835
Name	Cyproterone Acetate
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	9880
ChEBI	CHEBI:50743
ATC	G03HA01
Molecular formula	C24H29ClO4
Molecular weight	416.9
InChIKey	UWFYSQMTEOIJJG-FDTZYFLXSA-N

SMILES: CC(=O)[C@]1(CC[C@@H]2[C@@]1(CC[C@H]3[C@H]2C=C(C4=CC(=O)[C@@H]5C[C@@H]5[C@]34C)Cl)C)OC(=O)C

IUPAC name: [(1S,2S,3S,5R,11R,12S,15R,16S)-15-acetyl-9-chloro-2,16-dimethyl-6-oxo-15-pentacyclo[9.7.0.02,8.03,5.012,16]octadeca-7,9-dienyl] acetate

ChEBI definition: A steroid ester resulting from the formal condensation of the carboxy group of acetic acid with the 17-hydroxy group of cyproterone. It is an antiandrogenic drug which has recently been recognized to promote the occurrence and growth of intracranial meningiomas.

Pharmacological roles (ChEBI): androgen antagonist, progestin, geroprotector.

Also known as: Androcur, Androcur 10, Ciproterona, Cyprostat, Cyproterone, Cyproterone 17.alpha.-acetate, Cyproterone acetate, Diane, NSC-81430, SH 714, SH-714, SH714

Patent coverage: 6,370 distinct patent families (24,671 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
AR	Androgen receptor	Antagonist	7.85		P10275

Broader ChEMBL bioactivity targets: 14 (assay-derived). Sample: Nuclear receptor ROR-gamma, Prelamin-A/C, Androgen receptor, Thyrotropin receptor, Glucocorticoid receptor, Progesterone receptor, Adenosine A2 receptor, Muscarinic acetylcholine receptor M2, Mu-type opioid receptor, Androgen receptor.

Bioactivity

ChEMBL activities: 27 potent at pChembl ≥ 5 of 33 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
P25099	9.12	Ki	0.76	nM	CHEMBL_ACT_1174205
P25099	8.89	Ki	1.3	nM	CHEMBL_ACT_457316
PGR	8.52	AC50	3	nM	CHEMBL_ACT_25204427
P25099	8.3	IC50	5	nM	CHEMBL_ACT_381733
AR	8.15	IC50	7.1	nM	CHEMBL_ACT_25828641
AR	8.05	AC50	9	nM	CHEMBL_ACT_25203494
AR	7.85	Ki	14	nM	CHEMBL_ACT_553263
AR	7.64	IC50	23	nM	CHEMBL_ACT_553259
P15207	7.51	Ki	31	nM	CHEMBL_ACT_7680815
P15207	7.33	IC50	47	nM	CHEMBL_ACT_7680814
NR3C1	7.32	AC50	48	nM	CHEMBL_ACT_25175852
NR3C1	6.99	Ki	102	nM	CHEMBL_ACT_7678627
P25099	6.9	IC50	125	nM	CHEMBL_ACT_381734
NR3C1	6.65	IC50	225	nM	CHEMBL_ACT_7678626
AR	6.59	IC50	258.6	nM	CHEMBL_ACT_24406818
P29276	6.47	Ki	336	nM	CHEMBL_ACT_1174206
P15207	6.23	AC50	590	nM	CHEMBL_ACT_25187456
P15207	6.2	IC50	631	nM	CHEMBL_ACT_1605645
P29276	6.2	Ki	636	nM	CHEMBL_ACT_457317
OPRM1	6.05	Ki	886	nM	CHEMBL_ACT_7680733
P51450	5.85	Potency	1412	nM	CHEMBL_ACT_4770627
OPRM1	5.66	IC50	2183	nM	CHEMBL_ACT_7680732
CYP3A4	5.6	Potency	2512	nM	CHEMBL_ACT_4949240
CYP3A4	5.6	Potency	2512	nM	CHEMBL_ACT_5078589
AR	5.4	EC50	4000	nM	CHEMBL_ACT_26151149
CHRM2	5.04	AC50	9170	nM	CHEMBL_ACT_25195872
PGR	5.01	AC50	9780	nM	CHEMBL_ACT_25192808

Target pathways

Aggregated over 1 target gene(s): AR.

Top Reactome pathways

23 total, by targets touching each:

Pathway	Targets	Genes
Signal Transduction	1	AR
Signaling by Rho GTPases	1	AR
RHO GTPase Effectors	1	AR
Generic Transcription Pathway	1	AR
Cellular responses to stress	1	AR
SUMOylation	1	AR
SUMO E3 ligases SUMOylate target proteins	1	AR
HSP90 chaperone cycle for steroid hormone receptors (SHR) in the presence of ligand	1	AR
Nuclear Receptor transcription pathway	1	AR
Metabolism of proteins	1	AR
SUMOylation of intracellular receptors	1	AR
RHO GTPases activate PKNs	1	AR
Activated PKN1 stimulates transcription of AR (androgen receptor) regulated genes KLK2 and KLK3	1	AR
Deubiquitination	1	AR
Ub-specific processing proteases	1	AR
Post-translational protein modification	1	AR
RNA Polymerase II Transcription	1	AR
Gene expression (Transcription)	1	AR
Transcriptional regulation by RUNX2	1	AR
RUNX2 regulates osteoblast differentiation	1	AR
RUNX2 regulates bone development	1	AR
Cellular responses to stimuli	1	AR
Signaling by Rho GTPases, Miro GTPases and RHOBTB3	1	AR

Dominant GO biological processes

GO term	Targets
negative regulation of transcription by RNA polymerase II	1
MAPK cascade	1
in utero embryonic development	1
regulation of systemic arterial blood pressure	1
epithelial cell morphogenesis	1
transcription by RNA polymerase II	1
signal transduction	1
cell-cell signaling	1
spermatogenesis	1
single fertilization	1
positive regulation of cell population proliferation	1
negative regulation of cell population proliferation	1
male gonad development	1
positive regulation of gene expression	1
male somatic sex determination	1

Indications & clinical

Indications

8 indications (0 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
prostate adenocarcinoma	3	MONDO:0005082	EFO:0000673
endometriosis	3	MONDO:0005133	EFO:0001065
acne	3	MONDO:0011438	EFO:0003894
polycystic ovary syndrome	2	MONDO:0008487	EFO:0000660
hyperplasia	0	MONDO:0005043	EFO:0000536

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

Clinical trials

Total trials: 29.

Phase distribution

Phase	Trials
Not specified	13
PHASE3	8
PHASE4	5
PHASE2	2
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT01065220	PHASE4	COMPLETED	Sex Steroids and the Serotonin Transporter
NCT01103518	PHASE4	UNKNOWN	Ethinyl Estradiol and Cyproterone Acetate in Irregular Menstruation
NCT02027337	PHASE4	UNKNOWN	Hemocoagulation and Lipoperoxidation in Women Using Combined Oral Contraceptives, Correction by Antioxidants
NCT02715232	PHASE4	UNKNOWN	Effects of Sex Steroids on the Serotonin System
NCT02866786	PHASE4	COMPLETED	The Effects of OCP and Metformin on Clinical, Hormonal, Metabolic and Ultrasonographic Characteristics in PCOS
NCT00003653	PHASE3	COMPLETED	Hormone Therapy in Treating Patients With Rising PSA Levels Following Radiation Therapy for Prostate Cancer
NCT00005623	PHASE3	COMPLETED	Cyproterone Acetate in Treating Hot Flashes Following Surgical or Chemical Castration for Prostate Cancer
NCT00280657	PHASE3	COMPLETED	Study on Safety and Efficacy of an Oral Contraceptive in Treating Acne Papulopustulosa
NCT00363285	PHASE3	UNKNOWN	Cyproterone Acetate in Treating Patients With Newly Diagnosed Stage III or Stage IV Prostate Cancer
NCT00601276	PHASE3	TERMINATED	Comparison of Two Pharmacological Treatments of Pedophilia
NCT00849082	PHASE3	COMPLETED	Radiation Therapy With or Without Goserelin and Cyproterone in Treating Patients With Prostate Cancer
NCT01011751	PHASE3	COMPLETED	Treatment of Hot Flushes Caused by Leuprorelin 11.25 mg in Prostate Adenocarcinoma
NCT01973816	PHASE3	COMPLETED	MEdical Versus SUrgical Treatments of Rectal Endometriosis
NCT00196339	PHASE2	COMPLETED	A Clinical Trial to Study DR-2031 for the Treatment of Hot Flashes in Prostate Cancer Patients
NCT02729545	PHASE2	COMPLETED	The Efficacy and Safety Study of Tung’s Acupuncture for Polycystic Ovarian Syndrome
NCT04848181	EARLY_PHASE1	COMPLETED	The Effect of Pre-operative Use of Finasteride Versus Cyproterone Acetate on Blood Loss With Transurethral Resection of Prostate
NCT00908674	Not specified	COMPLETED	Androcur Effects on Quality of Life
NCT00919022	Not specified	TERMINATED	Androcur Non-interventional Study Among Patients With Carcinoma of the Prostate
NCT01573377	Not specified	COMPLETED	Efficacy of Metformin and Diane-35 on PCOS Patients,a Randomized, Controlled, Prospective Clinical Trial
NCT02349399	Not specified	COMPLETED	Drug Utilization Study on Diane-35 (and Generics) in Three European Healthcare Databases
NCT02410031	Not specified	COMPLETED	Risk Minimisation Study for Diane-35 and Its Generics
NCT02460445	Not specified	COMPLETED	Phlebotomy and Polycystic Ovary Syndrome
NCT02744131	Not specified	UNKNOWN	OCP vs Metformin for Improvement in Clinical Symptoms and Metabolic Markers in Indian PCOS Women
NCT03043924	Not specified	COMPLETED	Functional Study of the Hypothalamus in Magnetic Resonance Imaging (MRI) in Polycystic Ovary Syndrome (PCOS)
NCT04925180	Not specified	COMPLETED	A Study to Learn About the Awareness and Knowledge That Doctors Have About the Safety and Safe Use Information for Androcur and Other Cyproterone Acetate Treatments in Europe
NCT05021861	Not specified	UNKNOWN	Evaluation of Periodontal Status and hsCRP Levels in Females With PCOS on CPA/EE Combination Drug Regimen
NCT05657171	Not specified	COMPLETED	Effect of CPA/EE Drug on Periodontal Tissue and hsCRP Levels in PCOS Patients With Gingivitis
NCT07145281	Not specified	COMPLETED	Comparing the Effects of Sublingual Estradiol Treatment Versus Oral Estradiol With Cyproterone Acetate (CPA) Treatment on The Coagulation System in Transgender Women: A Prospective, Controlled Cohort Study
NCT07513480	Not specified	COMPLETED	Pretreatment With CPA-EE, Metformin and Myoinositol in PCOS With High AMH

Clinical evidence (CIViC)

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

Variant	Indication	Effect	Therapy	Level	CIViC
AR Mutation	Prostate Cancer	Resistance	Flutamide + Cyproterone Acetate + Nilutamide + Bicalutamide	CIViC B	EID1521

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.

129 molecules share ≥1 primary target. Top 60 by shared-target count:

Molecule	Source	Status	Shared targets
MEGESTROL	ChEMBL + PubChem	Phase 4 (approved)	AR
ABIRATERONE	ChEMBL	Phase 4 (approved)	AR
APALUTAMIDE	ChEMBL	Phase 4 (approved)	AR
ARIPIPRAZOLE	ChEMBL	Phase 4 (approved)	AR
BECLOMETHASONE DIPROPIONATE	ChEMBL	Phase 4 (approved)	AR
BETAMETHASONE	ChEMBL	Phase 4 (approved)	AR
BICALUTAMIDE	ChEMBL	Phase 4 (approved)	AR
BITHIONOL	ChEMBL	Phase 4 (approved)	AR
BROMHEXINE	ChEMBL	Phase 4 (approved)	AR
BUDESONIDE	ChEMBL	Phase 4 (approved)	AR
CHLORMADINONE	ChEMBL	Phase 4 (approved)	AR
CLARITHROMYCIN	ChEMBL	Phase 4 (approved)	AR
CLASCOTERONE	ChEMBL	Phase 4 (approved)	AR
CLOCORTOLONE PIVALATE	ChEMBL	Phase 4 (approved)	AR
CLOMIPHENE	ChEMBL	Phase 4 (approved)	AR
CORTISONE	ChEMBL	Phase 4 (approved)	AR
CYCLOFENIL	ChEMBL	Phase 4 (approved)	AR
DAROLUTAMIDE	ChEMBL	Phase 4 (approved)	AR
DESOGESTREL	ChEMBL	Phase 4 (approved)	AR
DESOXIMETASONE	ChEMBL	Phase 4 (approved)	AR
DEXAMETHASONE	ChEMBL	Phase 4 (approved)	AR
DIETHYLSTILBESTROL	ChEMBL	Phase 4 (approved)	AR
DIFLORASONE DIACETATE	ChEMBL	Phase 4 (approved)	AR
DORZOLAMIDE	ChEMBL	Phase 4 (approved)	AR
DROSPIRENONE	ChEMBL	Phase 4 (approved)	AR
DYDROGESTERONE	ChEMBL	Phase 4 (approved)	AR
ENZALUTAMIDE	ChEMBL	Phase 4 (approved)	AR
EPLERENONE	ChEMBL	Phase 4 (approved)	AR
ESTRADIOL	ChEMBL	Phase 4 (approved)	AR
ESTRADIOL CYPIONATE	ChEMBL	Phase 4 (approved)	AR
ESTRADIOL VALERATE	ChEMBL	Phase 4 (approved)	AR
ESTRIOL	ChEMBL	Phase 4 (approved)	AR
ESTRONE	ChEMBL	Phase 4 (approved)	AR
ETHINYL ESTRADIOL	ChEMBL	Phase 4 (approved)	AR
ETHYNODIOL DIACETATE	ChEMBL	Phase 4 (approved)	AR
ETONOGESTREL	ChEMBL	Phase 4 (approved)	AR
FLUMETHASONE PIVALATE	ChEMBL	Phase 4 (approved)	AR
FLUOCINOLONE ACETONIDE	ChEMBL	Phase 4 (approved)	AR
FLUOCINONIDE	ChEMBL	Phase 4 (approved)	AR
FLUOXYMESTERONE	ChEMBL	Phase 4 (approved)	AR
FLURANDRENOLIDE	ChEMBL	Phase 4 (approved)	AR
FLUTAMIDE	ChEMBL	Phase 4 (approved)	AR
FLUTICASONE FUROATE	ChEMBL	Phase 4 (approved)	AR
FLUTICASONE PROPIONATE	ChEMBL	Phase 4 (approved)	AR
HALCINONIDE	ChEMBL	Phase 4 (approved)	AR
HALOBETASOL PROPIONATE	ChEMBL	Phase 4 (approved)	AR
HEXACHLOROPHENE	ChEMBL	Phase 4 (approved)	AR
HEXESTROL	ChEMBL	Phase 4 (approved)	AR
HYDROCORTISONE	ChEMBL	Phase 4 (approved)	AR
INDOMETHACIN	ChEMBL	Phase 4 (approved)	AR
LEVONORGESTREL	ChEMBL	Phase 4 (approved)	AR
MEDROXYPROGESTERONE	ChEMBL	Phase 4 (approved)	AR
METHYLPREDNISOLONE	ChEMBL	Phase 4 (approved)	AR
MIFEPRISTONE	ChEMBL	Phase 4 (approved)	AR
MOMETASONE FUROATE	ChEMBL	Phase 4 (approved)	AR
NILUTAMIDE	ChEMBL	Phase 4 (approved)	AR
NOMEGESTROL	ChEMBL	Phase 4 (approved)	AR
NORETHINDRONE	ChEMBL	Phase 4 (approved)	AR
NORETHYNODREL	ChEMBL	Phase 4 (approved)	AR
OXANDROLONE	ChEMBL	Phase 4 (approved)	AR