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Protein / target

Steroid 17-alpha-hydroxylase/17,20 lyase

Encoded byCYP17A1P05093Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
3
Approved medicines
Open Targets target-level
View by indication →
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

Steroid 17-alpha-monooxygenase

Strongest disease association

Congenital adrenal hyperplasia due to 17-alpha-hydroxylase deficiency

Via encoding gene CYP17A1 · Genetic evidence · score 0.91

Therapeutic position

Established drug target

Small molecules

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 overview

A cytochrome P450 monooxygenase involved in corticoid and androgen biosynthesis.

View complete UniProt function annotation

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.

Approved medicines with mapped indications

3 medicines · 3 areas

Approved therapies targeting this protein, grouped by what they are approved to treat — the disease-first view of the medicines below. Relationships come from the canonical approved-indication graph (ChEMBL phase-4), the same source as the drug cards.

Cushing's Syndrome1 medicine
Prostatic Neoplasms1 medicine
Neoplasms1 medicine

Approved indications from ChEMBL (phase-4), via the canonical drug→disease graph.

Drugs targeting this protein

3

How approved and investigational drugs engage this protein — mechanism and action type, direct vs complex targeting, and how broadly each acts across other targets. A drug-first view (the section above groups the approved ones disease-first); direct binders with few recorded targets are listed first.

Abiraterone Acetate
Narrow target profileApprovedInhibitor

Cytochrome P450 17A1 inhibitor

Indicated for Prostatic Neoplasms

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

abiraterone
ApprovedInhibitor

Cytochrome P450 17A1 inhibitor

Indicated for Neoplasms

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

ketoconazole
Inhibitor

Cytochrome P450 17A1 inhibitor

Indicated for Cushing's Syndrome

Direct interaction with this protein · 1 of 4 recorded protein targets

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

Translational evidence

Open Targets 26

Why this target matters therapeutically, strongest evidence first. Disease associations are gene-level (via the gene that encodes this protein) and open into the full confidence synthesis; the development universe, tractability and safety annotations are target-level, from Open Targets.

Strongest disease associations · via encoding gene CYP17A1

Gene-level evidence surfaced through the gene CYP17A1that encodes this protein — not a direct protein–disease relationship. Ranked by Forefront's causal-directness weighting, so genetically- and clinically-evidenced diseases lead over ones that merely share the literature.

Congenital adrenal hyperplasia due to 17-alpha-hydroxylase deficiency
0.92Well supported

Genetic evidence dominant · Open Targets 0.80

Adrenal Hyperplasia, Congenital
0.91Well supported

Genetic evidence dominant · Open Targets 0.64

46,XY disorder of sex development due to isolated 17,20 lyase deficiency
0.89Well supported

Genetic evidence dominant · Open Targets 0.67

17-Alpha-hydroxylase/17,20-lyase deficiency, combined complete
0.87Well supported

Genetic evidence dominant · Open Targets 0.53

17-Alpha-hydroxylase/17,20-lyase deficiency, combined partial
0.85Well supported

Genetic evidence dominant · Open Targets 0.52

View evidence synthesis (5)
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

Adrenal Hyperplasia, CongenitalWell 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 per-type scores above show the calculation.

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
Adrenal Hyperplasia, Congenital0.64
Prostatic Neoplasms0.61
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's Syndrome0.49

Drug development

6 compounds recorded · 3 approved · 3 in clinical development

Open Targets' development universe — every compound recorded against the target at any stage, not all approved medicines. Distinct from the 3 drugs that target this protein in Forefront's canonical graph (3 with a mapped approved indication, shown above): these count different sets and are not a subset relation.

View all recorded compounds (6)
SEVITERONELPhase 2
LEVOKETOCONAZOLEApproval
ORTERONELPhase 3
GALETERONEPhase 3
ABIRATERONEApproval
ABIRATERONE ACETATEApproval

Open Targets known-drugs universe. Drug name and highest clinical stage only — the disease relationship is NOT read from this slice (it carries trial-context noise); approved indications come from the canonical graph above.

Tractability

Small moleculesStrong

Approved Drug and Structure with Ligand support this modality.

Protein degradersEmerging

Feasibility evidence (small molecule binder) — no clinical-stage drug of this modality recorded.

View underlying tractability evidence (5)
SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · Druggable FamilyPR · Small Molecule Binder

Raw Open Targets tractability assessment buckets, by modality.

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. Ranked by active status, then clinical phase and recency, and spread across the targeting drugs.

View all trials (26)

RECRUITING · via Abiraterone Acetate · NCT05593497

ACTIVE_NOT_RECRUITING · via Abiraterone Acetate · NCT05137262

ClinicalTrials.gov via the drug-target graph.

What's happening now

12

Recent therapeutic activity around this target — regulatory actions, clinical trials and safety signals for 3 drugs that target this protein, plus publications where such a drug is a genuine subject. Every item is reached indirectly through the drug, not the protein itself; incidental mentions (a paper that merely measures a drug) and press items are excluded.

  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. Regulatory approval2019-02-25

    Approval: ABIRATERONE ACETATE (ANDA208371)

    fda · regulatory · fda · via Abiraterone Acetate

  11. Accelerated approval granted2014-11-18

    Accelerated approval: Ketoconazole Esteve (previously Ketoconazole HRA) (EMA)

    ema · regulatory · ema · via ketoconazole

  12. Accelerated approval granted2011-09-05

    Accelerated approval: Zytiga (EMA)

    ema · regulatory · ema · 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.