FGFR1 Inhibitors
9 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.
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 |
|---|---|---|---|
| OFEV | Boehringer Ingelheim | 2014 | 3 |
| VOTRIENT | Novartis | 2009 | 2 |
| GAVRETO | RIGEL PHARMS | 2020 | 2 |
| PAZOPANIB HYDROCHLORIDE | EUGIA PHARMA | 2023 | 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 847 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on FGFR1. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 246 | 158 | 34 | 53 | 82% |
| Phase 2 | 473 | 180 | 94 | 196 | 66% |
| Phase 3 | 106 | 51 | 15 | 40 | 77% |
| Phase 4 | 22 | 14 | 2 | 6 | 88% |
Top Sponsors
By Modality
Top Conditions
Phase 3 Readout Calendar Pro
4 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 (2009 - 2023)
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
- • 9 companies competing
- • Market share by company
Full Drug Portfolio
- • All 9 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 9-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 315 (74 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 FGFR1. A trial counts if an approved drug acting on FGFR1 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.