FGFR2 Inhibitors
6 drugsAbout FGFR2
Fibroblast growth factor receptor 2 (FGFR2) is a receptor tyrosine kinase involved in cell proliferation, differentiation, and angiogenesis signaling pathways.
FGFR2 is strongly supported by human genetics (max score 0.97), with variants linked to Pfeiffer syndrome (0.97), Crouzon syndrome (0.95), and FGFR2-related craniosynostosis (0.94). Activation of FGFR2 is likely beneficial based on genetic evidence.
Six FDA-approved drugs target FGFR2, including LENVIMA, RETEVMO, STIVARGA, OFEV, and PEMAZYRE, all of which are small molecules. These drugs span oncology, respiratory, and other therapeutic areas.
Strategic Insights
ℹ️ How we calculate- Validated target with strong trial activity and 81% attractiveness score.
Human Genetic Evidence Strong
FGFR2 has strong genetic support with a maximum score of 0.97 across 57 diseases.
Strong genetic support suggests FGFR2-targeting therapies have a higher probability of clinical success.
💡 Why activation?
- • Loss-of-function variants increase disease risk (OR > 1) — restoring function may help
- • 50% directional consistency across 2 traits
- • Strong signal in genetic, familial or congenital disease, musculoskeletal or connective tissue disease, phenotype pathways
Cross-Disease Effects
Trade-off: HighDirection of Effect
50% alignedEvidence Across Diseases
20 totalGWAS and other genetic studies link FGFR2 to 57 diseases.
Gain-of-function causes disease; inhibition may help
🔗 Colocalization Evidence 20 strong
max H4: 1.00eQTL/pQTL signals for FGFR2 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 Drugs
Six companies have approved FGFR2-targeting drugs, including EISAI INC, INCYTE CORP, and Eli Lilly.
The presence of multiple companies indicates a moderately competitive landscape with potential entry barriers.
| Drug | Company | Approved | Indications |
|---|---|---|---|
| OFEV | Boehringer Ingelheim | 2014 | 3 |
| PEMAZYRE | INCYTE CORP | 2020 | 2 |
| BALVERSA | Johnson & Johnson | 2019 | 1 |
Drug Modality Landscape
Modalities
Routes of Administration
FGFR2 is amenable to small molecule drugs, with oral options available for convenient dosing.
The absence of other modalities like antibodies or biologics represents a potential whitespace opportunity.
Clinical Trials 860 trials
Completion by Phase
| Phase | Total | Completed | Failed | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 230 | 139 | 22 | 68 | 86% |
| Phase 2 | 455 | 137 | 72 | 240 | 66% |
| Phase 3 | 145 | 81 | 15 | 49 | 84% |
| Phase 4 | 30 | 16 | 3 | 11 | 84% |
Top Sponsors
By Modality
Top Conditions
Phase 3 Readout Calendar Pro
6 Phase 3 trials testing approved FGFR2 drugs across all sponsors.
Coverage: trials whose intervention is an approved drug targeting FGFR2. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.
Drug Approval Timeline (2012 - 2020)
The first FGFR2-targeting drug was approved in 2012 (STIVARGA), with the most recent approval in 2020 (PEMAZYRE).
The approval timeline suggests a relatively stable market with potential for further innovation.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 6 companies competing
- • Market share by company
Full Drug Portfolio
- • All 6 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 6-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Competition: High (15 sponsors)
- • 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 735 clinical trials targeting FGFR2.
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