Protein / target

Steroid 17-alpha-hydroxylase/17,20 lyase

CYP17A1P05093Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
3
Approved medicines
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

Steroid 17-alpha-monooxygenase activity

Primary system

Nervous system

Strongest disease association

congenital adrenal hyperplasia due to 17-alpha-hydroxylase deficiency

Genetic evidence · score 0.91

Therapeutic maturity

Clinically validated target

3 approved medicines against this target

Druggability

Small molecule

Open Targets tractability · Approved Drug

Clinical development

3 approved · 3 in clinical development

30 linked trials

Derived from structured UniProt, Open Targets and literature data on this page.

Protein profile

UniProt 2026_02

Canonical identity and biological annotation from UniProt.

Function

A cytochrome P450 monooxygenase involved in corticoid and androgen biosynthesis (PubMed:22266943, PubMed:25301938, PubMed:27339894, PubMed:9452426). Catalyzes 17-alpha hydroxylation of C21 steroids, which is common for both pathways. A second oxidative step, required only for androgen synthesis, involves an acyl-carbon cleavage. The 17-alpha hydroxy intermediates, as part of adrenal glucocorticoids biosynthesis pathway, are precursors of cortisol (Probable) (PubMed:25301938, PubMed:9452426). Hydroxylates steroid hormones, pregnenolone and progesterone to form 17-alpha hydroxy metabolites, followed by the cleavage of the C17-C20 bond to form C19 steroids, dehydroepiandrosterone (DHEA) and androstenedione (PubMed:22266943, PubMed:25301938, PubMed:27339894, PubMed:36640554, PubMed:9452426). Has 16-alpha hydroxylase activity. Catalyzes 16-alpha hydroxylation of 17-alpha hydroxy pregnenolone, followed by the cleavage of the C17-C20 bond to form 16-alpha-hydroxy DHEA (PubMed:36640554). Also 16-alpha hydroxylates androgens, relevant for estriol synthesis (PubMed:25301938, PubMed:27339894). Mechanistically, uses molecular oxygen inserting one oxygen atom into a substrate, and reducing the second into a water molecule, with two electrons provided by NADPH via cytochrome P450 reductase (CPR; NADPH-ferrihemoprotein reductase) (PubMed:22266943, PubMed:25301938, PubMed:27339894, PubMed:9452426)

Subcellular location

Endoplasmic reticulum membraneMicrosome membrane
Domains and Gene Ontology detail (17)

Gene Ontology

  • Caxon
  • Cendoplasmic reticulum
  • Cendoplasmic reticulum membrane
  • Cneuronal cell body
  • Fheme binding
  • Firon ion binding
  • Flyase activity
  • Foxygen binding
  • Fsteroid 17-alpha-monooxygenase activity
  • Pandrogen biosynthetic process
  • Pcortisol biosynthetic process
  • Pglucocorticoid biosynthetic process

508 aa · 57 kDa

Biological roles

Reactome v97

What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.

Metabolic enzyme activityGO
View supporting evidence

Metabolic enzyme activity

  • ·progesterone metabolic process
  • ·steroid metabolic process
View underlying pathways (3)

Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.

Interaction neighbourhood

STRING v12.0

Proteins with the strongest functional or physical association. Node size and line weight reflect STRING confidence; hover or select a partner to inspect one association.

HSD3B1HSD3B2SRD5A1HSD17B3SRD5A2CYB5BCYB5AAKR1C3HSD17B2CYP19A1CYP17A1

10 strongest partners — larger node and heavier line mean higher confidence

Functional and physical associations from STRING v12.0. Only associations with this protein are drawn — partner-to-partner links are not part of this evidence.

Drugs targeting this protein

2

Whether each drug engages this protein directly or through a complex, and how many other targets are recorded for it. Direct binders with few recorded targets are listed first.

Abiraterone Acetate
Narrow target profileApprovedInhibitor

Cytochrome P450 17A1 inhibitor

Appears in clinical studies involving prostate cancer, prostate carcinoma, prostate neoplasm, prostate cancer

Direct interaction with this protein · Only this protein recorded as a target

abiraterone
ApprovedInhibitor

Cytochrome P450 17A1 inhibitor

Appears in clinical studies involving prostate cancer, prostate neoplasm, neoplasm, cancer

Direct interaction with this protein · 1 of 5 recorded protein targets

ChEMBL mechanism, action type and target identity. Open Targets clinical status and indications.

Translational evidence

Open Targets 26

Why this target matters therapeutically — disease associations, drugs in development, tractability and safety, from Open Targets.

Strongest genetic associations

Human genetic evidence — the most direct causal link between this target and a disease.

congenital adrenal hyperplasia due to 17-alpha-hydroxylase deficiency0.91

Genetic · overall 0.80

congenital adrenal hyperplasia0.89

Genetic · overall 0.64

46,XY disorder of sex development due to isolated 17,20 lyase deficiency0.88

Genetic · overall 0.67

17-alpha-hydroxylase/17,20-lyase deficiency, combined complete0.87

Genetic · overall 0.53

17-alpha-hydroxylase/17,20-lyase deficiency, combined partial0.85

Genetic · overall 0.52

Highest-confidence therapeutic associations

Diseases where a drug acting on this target has already reached clinical development.

prostate cancer0.97

Clinical · overall 0.61

prostate neoplasm0.83

Clinical · overall 0.50

Cushing syndrome0.79

Clinical · overall 0.49

adrenal gland disorder0.61

Clinical · overall 0.60

Highest overall evidence

Remaining associations by Open Targets' aggregated evidence score.

17,20-lyase deficiency, isolated0.57

Genetic literature

Show all associations
congenital adrenal hyperplasia due to 17-alpha-hydroxylase deficiency0.80
46,XY disorder of sex development due to isolated 17,20 lyase deficiency0.67
congenital adrenal hyperplasia0.64
prostate cancer0.61
adrenal gland disorder0.60
17,20-lyase deficiency, isolated0.57
17-alpha-hydroxylase/17,20-lyase deficiency, combined complete0.53
17-alpha-hydroxylase/17,20-lyase deficiency, combined partial0.52
prostate neoplasm0.50
Cushing syndrome0.49

Open Targets ranks 570 associations for this target and we store the top 10 by aggregated score. The long tail is largely literature co-mention and RNA-expression evidence, not evidence that this target drives those diseases.

Known drugs · 6 total

SEVITERONELPhase 2

breast cancer · prostate cancer · triple-negative breast carcinoma

LEVOKETOCONAZOLEApproval

Cushing syndrome · adrenal gland hyperfunction · adrenal gland disorder

ORTERONELPhase 3

prostate cancer · breast cancer · ovarian granulosa cell tumor

GALETERONEPhase 3

prostate cancer · pancreatic adenocarcinoma

ABIRATERONEApproval

prostate cancer · prostate neoplasm · neoplasm

ABIRATERONE ACETATEApproval

prostate cancer · prostate carcinoma · prostate neoplasm

Tractability

SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · Druggable FamilyPR · Small Molecule Binder

Clinical trials

30

Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein.

Show remaining trials (24)

RECRUITING · via Abiraterone Acetate · NCT07001709

ACTIVE_NOT_RECRUITING · via Abiraterone Acetate · NCT05137262

TERMINATED · via Abiraterone Acetate · NCT03649841

ClinicalTrials.gov via the drug-target graph.

Forefront confidence

Synthesis

Our own weighting of the evidence behind each disease association. Human genetics and clinical evidence count for most; literature co-mention counts for little, because two entities sharing an abstract is not evidence that one drives the other.

congenital adrenal hyperplasia due to 17-alpha-hydroxylase deficiencyWell supported
0.92
agreement 0.801.00
Genetic87%Animal model10%Literature3%Genetic literaturedup

Open Targets aggregate 0.80 · 3 independent evidence families · 1 not counted as duplicate

congenital adrenal hyperplasiaWell supported
0.91
agreement 0.811.00
Genetic79%Animal model9%Clinical8%Literature3%Genetic literaturedup

Open Targets aggregate 0.64 · 4 independent evidence families · 1 not counted as duplicate

46,XY disorder of sex development due to isolated 17,20 lyase deficiencyWell supported
0.89
agreement 0.771.00
Genetic87%Animal model13%

Open Targets aggregate 0.67 · 2 independent evidence families

17-alpha-hydroxylase/17,20-lyase deficiency, combined completeWell supported
0.87
agreement 0.750.99
Genetic100%

Open Targets aggregate 0.53 · 1 independent evidence family

17-alpha-hydroxylase/17,20-lyase deficiency, combined partialWell supported
0.85
agreement 0.730.97
Genetic100%

Open Targets aggregate 0.52 · 1 independent evidence family

The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the underlying per-type scores are shown above so the calculation can be checked.

What's happening now

18

Recent activity around this target, drawn from one canonical event stream. Every item is reached through 2 drugs that target this protein, so each event is news about that drug rather than about the protein directly.

  1. Label change2026-06-05

    Label change: ABIRATERONE ACETATE (ANDA208371)

    fda · regulatory · fda · via Abiraterone Acetate

  2. New publication2025-10-07
    Niraparib and abiraterone acetate plus prednisone for HRR-deficient metastatic castration-sensitive prostate cancer: a randomized phase 3 trial.

    Nature medicine · 2025 · 19 citations · Europe PMC · via Abiraterone Acetate

  3. New publication2024-12-11
    AR alterations inform circulating tumor DNA detection in metastatic castration resistant prostate cancer patients.

    Nature communications · 2024 · 11 citations · Europe PMC · via abiraterone

  4. New publication2024-12-01
    Luteinizing hormone-releasing hormone receptor agonists and antagonists in prostate cancer: effects on long-term survival and combined therapy with next-generation hormonal agents.

    Cancer biology & medicine · 2024 · 9 citations · Europe PMC · via abiraterone

  5. New publication2024-09-01
    Circulating Tumor DNA Assessment for Treatment Monitoring Adds Value to PSA in Metastatic Castration-Resistant Prostate Cancer.

    Clinical cancer research : an official journal of the American Association for Cancer Research · 2024 · 19 citations · Europe PMC · via abiraterone

  6. New publication2023-02-16
    Rucaparib or Physician's Choice in Metastatic Prostate Cancer.

    The New England journal of medicine · 2023 · 332 citations · Europe PMC · via abiraterone

  7. Regulatory approval2021-08-20

    Approval: Abiraterone Mylan (EMA)

    ema · regulatory · ema · via abiraterone

  8. Regulatory approval2021-06-24

    Approval: Abiraterone Krka (EMA)

    ema · regulatory · ema · via abiraterone

  9. Regulatory approval2021-04-26

    Approval: Abiraterone Accord (EMA)

    ema · regulatory · ema · via abiraterone

  10. New publication2020-04-28
    Olaparib for Metastatic Castration-Resistant Prostate Cancer.

    The New England journal of medicine · 2020 · 1,688 citations · Europe PMC · via abiraterone

  11. New publication2019-09-30
    Cabazitaxel versus Abiraterone or Enzalutamide in Metastatic Prostate Cancer.

    The New England journal of medicine · 2019 · 439 citations · Europe PMC · via abiraterone

  12. New publication2019-05-06
    Genomic correlates of clinical outcome in advanced prostate cancer.

    Proceedings of the National Academy of Sciences of the United States of America · 2019 · 1,163 citations · Europe PMC · via abiraterone

Objective event titles are shown unmodified; the event kind and significance line are derived from structured fields. Forefront AttentionEvent stream aggregating Europe PMC Regulatory filings ClinicalTrials.gov.