FGFR Inhibitors
1 drugsAbout FGFR
Fibroblast Growth Factor Receptors (FGFRs) are receptor tyrosine kinases mediating cell signaling for growth, differentiation, angiogenesis, and wound healing. Activated by fibroblast growth factors (FGFs), they trigger intracellular cascades influencing cell behavior and tissue repair.
FGFR1 has strong genetic support as a therapeutic target (score 0.98), with loss-of-function variants linked to hypogonadotropic hypogonadism and other syndromes. Activation of FGFR1 is likely beneficial based on genetic evidence.
ICLUSIG (ponatinib) is the only FDA-approved drug targeting FGFR, developed by Takeda as a small molecule. It has two indications and was first approved in 2012.
Strategic Insights
ℹ️ How we calculate- White space opportunity in B Acute Lymphoblastic Leukemia, Philadelphia Chromosome Negative with only 1 trials.
- phase1 represents biological uncertainty with 50% completion.
Human Genetic Evidence Strong
FGFR1 has strong genetic support with a max score of 0.98 across 26 diseases.
Strong genetic support suggests FGFR1-targeting therapies have a higher 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 genetic, familial or congenital disease, endocrine system disease, phenotype pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
20 totalGWAS and other genetic studies link FGFR1 to 26 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 FGFR1 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
Takeda is the only company with an approved FGFR-targeting drug.
The market is highly concentrated, indicating potential entry barriers for new competitors.
Drug Modality Landscape
Modalities
Routes of Administration
Only one approved drug targets FGFR, using small molecule modality.
The lack of diverse modalities represents a whitespace opportunity for novel FGFR-targeting approaches.
Clinical Trials 56 trials
Completion by Phase
| Phase | Total | Completed | Failed | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 17 | 10 | 4 | 3 | 71% |
| Phase 2 | 32 | 10 | 9 | 12 | 53% |
| Phase 3 | 5 | 1 | 1 | 3 | 50% |
| Phase 4 | 2 | 1 | 1 | 0 | 50% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
Drug Approval Timeline (2012 - 2012)
Philadelphia Chromosome-Positive Acute Lymphoblastic Leuk...
ICLUSIG was approved in 2012 and remains the most recent and only approved drug.
The approval timeline suggests a potential saturation or lack of recent innovation in FGFR-targeting therapies.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 1 companies competing
- • Market share by company
Full Drug Portfolio
- • All 1 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 1-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 35 clinical trials targeting FGFR.
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