CYP17 Inhibitors
4 drugsAbout CYP17
CYP17 (cytochrome P450 17A1) is a crucial enzyme in steroid hormone biosynthesis, catalyzing reactions for androgen, estrogen, and cortisol production. Its activity is essential for hormone-driven processes, making it a significant drug target.
Human genetic studies provide strong validation for CYP17 as a therapeutic target (max score 0.91), with loss-of-function variants linked to congenital adrenal hyperplasia. Activation of CYP17 is likely beneficial based on genetic evidence.
CYP17 is targeted by 4 FDA-approved small molecule drugs including ABIRATERONE ACETATE, ZYTIGA, AKEEGA and YONSA, primarily for oncology and other indications.
CYP17 Genetic Evidence Strong
Strong genetic evidence supports CYP17's role in congenital adrenal hyperplasia (score 0.91).
High genetic support suggests that CYP17-targeting therapies have an increased probability of clinical success.
💡 Why activation?
- • Loss-of-function variants increase disease risk (OR > 1) — restoring function may help
- • 100% directional consistency across 3 traits
- • Strong signal in reproductive system or breast disease, endocrine system disease, nutritional or metabolic disease pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
20 totalGWAS and other genetic studies link CYP17A1 to 22 diseases.
Loss-of-function causes disease; activation may help
Loss-of-function causes disease; activation may help
🔗 Colocalization Evidence 20 strong
max H4: 1.00eQTL/pQTL signals for CYP17A1 colocalize with these GWAS traits, providing causal evidence that gene expression changes drive disease risk.
Understanding these scores
Association Score (0-1): Combines all evidence types (genetic, literature, drugs, animal models). Higher = more evidence linking target to disease. This is a ranking heuristic, not a confidence score.
L2G Score: Open Targets uses a machine learning model (Locus-to-Gene) to predict which gene is causal at each GWAS locus. L2G=0.5 means ~50% probability of being the causal gene. Only associations with L2G > 0.05 are included.
Odds Ratio (OR): From gene burden studies (UK Biobank, AstraZeneca PheWAS). Measures how loss-of-function variants affect disease risk. OR<1 = protective (inhibiting target may help), OR>1 = risk (losing function causes disease).
Beta (β): Effect size for continuous traits. β<0 = protective, β>0 = risk.
Clinical Translation (~1.8x): Based on Nelson et al. 2015: drug targets with genetic evidence have ~2x higher success rates in clinical trials. We estimate: Strong support (score ≥0.7) → ~1.8x, Moderate (0.3-0.7) → ~1.3x, Weak → baseline.
Colocalization (H4): Tests whether a GWAS signal and an eQTL/pQTL signal share the same causal variant. H4 is the posterior probability that both traits are associated AND share a causal variant. H4 > 0.8 = strong evidence that gene expression/protein levels drive disease risk. This links genetic variation → gene expression → disease, supporting the target-disease connection.
Top CYP17 Drugs
Three companies, Apotex, Johnson & Johnson, and Sun Pharma, have approved CYP17-targeting drugs.
The market is relatively concentrated, suggesting potential entry barriers for new companies.
| Drug | Company | Approved | Indications |
|---|---|---|---|
| YONSA | Sun Pharma | 2018 | 1 |
CYP17 Drug Modality Landscape
Modalities
Routes of Administration
CYP17 is amenable to small molecule drugs, with oral options available for convenient dosing.
Exploring alternative modalities like antibodies or PROTACs could provide a competitive advantage.
CYP17 Clinical Trials 389 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on CYP17. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 124 | 53 | 33 | 38 | 62% |
| Phase 2 | 200 | 83 | 40 | 76 | 67% |
| Phase 3 | 56 | 17 | 3 | 36 | 85% |
| Phase 4 | 9 | 4 | 0 | 5 | 100% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
Phase 3 Readout Calendar Pro
4 Phase 3 trials testing approved CYP17 drugs across all sponsors.
Coverage: trials whose intervention is an approved drug targeting CYP17. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.
CYP17 Drug Approval Timeline (2011 - 2023)
The first CYP17-targeting drug was approved in 2011, with the most recent in 2023.
The approval timeline indicates continued interest in CYP17 as a target, though the rate is slowing.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 3 companies competing
- • Market share by company
Full Drug Portfolio
- • All 4 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 4-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 192 (63 companies)
- • Completion rates by condition
Full summary • All drugs • Genetic evidence • Trials • Timeline
How We Count Trials
- Trials: phase 1-4 interventional trials on ClinicalTrials.gov that started in 2008 or later and test an FDA-approved drug acting on CYP17. A trial counts if an approved drug acting on CYP17 is in any arm, including as a comparator, and a trial that tests two such drugs counts once. Trials that test only unapproved drugs are not counted. A trial listed as phase 1/2 counts as phase 1, and phase 2/3 as phase 2.
- Active: recruiting, not yet recruiting, enrolling by invitation, or active and no longer recruiting.
- Completion rate: of the trials that have ended, the share marked Completed rather than Terminated or Withdrawn. Completed means the trial ended as planned; it does not mean the drug worked. Suspended trials are not counted as ended.
- Stopped: terminated or withdrawn. Trials stop for many reasons, including slow enrolment and business decisions, not only safety or efficacy.
- Sponsors: the lead sponsor of each trial. Companies are the sponsors ClinicalTrials.gov classes as industry; the rest are universities, hospitals, cooperative groups, government bodies and individuals.