FGFR1 Inhibitors
7 drugsAbout FGFR1
Fibroblast growth factor receptor 1 (FGFR1) is a receptor tyrosine kinase crucial for cell signaling pathways involved in cell growth, differentiation, and survival. It plays a key role in cell communication and regulation of various biological processes.
Human genetics provide strong validation for FGFR1 as a therapeutic target (max score 0.98), with loss-of-function variants linked to diseases like hypogonadotropic hypogonadism. Activation of FGFR1 is likely beneficial based on genetic evidence.
FGFR1 is targeted by 7 FDA-approved small molecule drugs including LENVIMA, RETEVMO and PEMAZYRE, spanning oncology and respiratory indications. Several companies have approved drugs, including EISAI INC, INCYTE CORP and Eli Lilly.
Strategic Insights
ℹ️ How we calculate- Validated target with strong trial activity and 81% attractiveness score.
FGFR1 Genetic Evidence Strong
FGFR1 has strong genetic support with a maximum score of 0.98 linking it to 26 diseases.
Strong genetic support suggests FGFR1-targeting therapies have a higher likelihood 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 FGFR1 Drugs
Seven companies have approved FGFR1-targeting drugs, with EISAI INC, INCYTE CORP, and Eli Lilly as top players.
The presence of multiple companies indicates a competitive market, requiring differentiated strategies for new entrants.
| Drug | Company | Approved | Indications |
|---|---|---|---|
| STIVARGA | Bayer | 2012 | 3 |
| GAVRETO | RIGEL PHARMS | 2020 | 2 |
| PEMAZYRE | INCYTE CORP | 2020 | 2 |
| BALVERSA | Johnson & Johnson | 2019 | 1 |
FGFR1 Drug Modality Landscape
Modalities
Routes of Administration
FGFR1 is amenable to small molecule drugs, with oral options available for convenient dosing.
The absence of other modalities like antibodies represents a whitespace opportunity for novel FGFR1 therapies.
FGFR1 Clinical Trials 765 trials
Completion by Phase
| Phase | Total | Completed | Failed | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 210 | 126 | 22 | 61 | 85% |
| Phase 2 | 435 | 124 | 70 | 236 | 64% |
| Phase 3 | 99 | 44 | 13 | 42 | 77% |
| Phase 4 | 21 | 11 | 2 | 8 | 85% |
Top Sponsors
By Modality
Top Conditions
Phase 3 Readout Calendar Pro
5 Phase 3 trials testing approved FGFR1 drugs across all sponsors.
Coverage: trials whose intervention is an approved drug targeting FGFR1. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.
FGFR1 Drug Approval Timeline (2012 - 2020)
The first FGFR1-targeting drug was approved in 2012 (STIVARGA), with the most recent approval in 2020 (GAVRETO).
The approval timeline suggests a maturing market, potentially indicating a shift towards incremental innovation.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 7 companies competing
- • Market share by company
Full Drug Portfolio
- • All 7 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 7-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 743 clinical trials targeting FGFR1.
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