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.
FGFR 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 FGFR Drugs
Takeda is the only company with an approved FGFR-targeting drug.
The market is highly concentrated, indicating potential entry barriers for new competitors.
FGFR 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.
FGFR Clinical Trials 43 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on FGFR. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 8 | 2 | 4 | 2 | 33% |
| Phase 2 | 32 | 11 | 9 | 12 | 55% |
| Phase 3 | 3 | 0 | 1 | 2 | 0% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
FGFR 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
- • Sponsors: 23 (4 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 FGFR. A trial counts if an approved drug acting on FGFR 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.