Carbonic anhydrase Inhibitors
11 drugsAbout Carbonic anhydrase
Carbonic anhydrase catalyzes the interconversion of carbon dioxide and water to bicarbonate and protons. It is involved in various physiological processes, making it relevant to multiple therapeutic areas.
Human genetic studies provide strong validation for targeting CA2 (max score 0.87). Loss-of-function variants are associated with osteopetrosis and renal tubular acidosis, suggesting activation may be beneficial.
Carbonic anhydrase is targeted by 11 FDA-approved small molecule drugs, including TOPIRAMATE and EPRONTIA. These drugs are used in CNS disorders, metabolic conditions, and ophthalmology.
Strategic Insights
ℹ️ How we calculate- White space opportunity in Weight Loss with only 2 trials.
Carbonic anhydrase Genetic Evidence Strong
Genetic evidence strongly supports CA2 as a drug target (max score 0.87).
Strong genetic support increases confidence in target validation and clinical development.
💡 Why activation?
- • Loss-of-function variants increase disease risk (OR > 1) — restoring function may help
- • 100% directional consistency across 3 traits
- • Strong signal in urinary system disease, genetic, familial or congenital disease, musculoskeletal or connective tissue disease pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
5 totalGWAS and other genetic studies link CA2 to 5 diseases.
Loss-of-function causes disease; activation may help
Loss-of-function causes disease; activation may help
🔗 Colocalization Evidence 19 strong
max H4: 0.99eQTL/pQTL signals for CA2 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 Carbonic anhydrase Drugs
Ten companies have approved drugs targeting carbonic anhydrase.
The presence of multiple players indicates a competitive market with moderate entry barriers.
Carbonic anhydrase Drug Modality Landscape
Modalities
Routes of Administration
Carbonic anhydrase is amenable to small molecule drugs, with oral options available for convenient dosing.
Exploring alternative modalities like biologics could provide a competitive advantage.
Carbonic anhydrase Clinical Trials 199 trials
Completion by Phase
| Phase | Total | Completed | Failed | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 25 | 21 | 3 | 1 | 88% |
| Phase 2 | 56 | 33 | 16 | 7 | 67% |
| Phase 3 | 47 | 35 | 10 | 2 | 78% |
| Phase 4 | 71 | 48 | 8 | 14 | 86% |
Top Sponsors
By Modality
Top Conditions
Carbonic anhydrase Drug Approval Timeline (1958 - 2025)
The first drug was approved in 1958, and the most recent in 2025.
The long approval history suggests sustained interest and potential for further innovation.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 10 companies competing
- • Market share by company
Full Drug Portfolio
- • All 11 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 11-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Competition: High (15 sponsors)
- • White space: 10 underexplored indications
- • Success rates by condition
Full summary • All drugs • Genetic evidence • Trials • Timeline
How We Calculate These Metrics
Target Attractiveness Score
A 0-100 score based on trial activity, sponsor diversity, and completion rates. Calculated from 132 clinical trials targeting Carbonic anhydrase.
Completion rate: Percentage of trials that reached their planned endpoint. Trials terminated early, withdrawn, or suspended are not counted—these often indicate safety issues, lack of efficacy, or strategic pivots.
- Highly Attractive (80+): High trial activity, many sponsors, strong completion rates
- Attractive (60-79): Good trial activity and validation
- Moderate (40-59): Moderate interest from sponsors
- Low (under 40): Limited trial activity or validation concerns
Strategic Insights
Auto-generated insights based on trial analytics including competition intensity, white space opportunities, modality shifts, and failure patterns. We analyze trial sponsors, phases, indications, and outcomes.
Risk Signals
- High Competition: Many sponsors competing for this target (may reduce market opportunity)
- High Failure Risk: Low trial completion rates suggest development challenges
- Low Validation: Limited trial activity or poor outcomes indicate uncertain viability
- White Space Available: Underexplored indications present opportunities