FGFR3 Inhibitors
8 drugsAbout FGFR3
Fibroblast growth factor receptor 3 (FGFR3) is a receptor tyrosine kinase involved in cell growth, differentiation, and survival signaling. As a key regulator of these processes, it has emerged as a target for drug development.
Human genetics provide strong validation for FGFR3 as a therapeutic target (max score 0.90). Loss-of-function variants are associated with diseases like thanatophoric dysplasia type 1 (score 0.90), achondroplasia (0.89), and cervical cancer (0.89), suggesting activation may be beneficial.
FGFR3 is targeted by five FDA-approved small molecule drugs, including LENVIMA, PEMAZYRE, RETEVMO, BALVERSA, and OFEV. These drugs span oncology and respiratory indications.
FGFR3 Genetic Evidence Strong
FGFR3 has strong genetic support with a maximum score of 0.90 across 59 diseases.
Strong genetic support suggests FGFR3-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, musculoskeletal or connective tissue disease, phenotype pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
20 totalGWAS and other genetic studies link FGFR3 to 59 diseases.
Loss-of-function causes disease; activation may help
Loss-of-function causes disease; activation may help
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 FGFR3 Drugs
Five companies have approved FGFR3-targeting drugs, including EISAI INC, INCYTE CORP, and Eli Lilly.
The presence of multiple companies suggests moderate competition, but also established market entry barriers.
| Drug | Company | Approved | Indications |
|---|---|---|---|
| VOTRIENT | Novartis | 2009 | 2 |
| PAZOPANIB HYDROCHLORIDE | EUGIA PHARMA | 2023 | 2 |
| PEMAZYRE | INCYTE CORP | 2020 | 2 |
| BALVERSA | Johnson & Johnson | 2019 | 1 |
| YUVIWEL | ASCENDIS | 2026 | - |
FGFR3 Drug Modality Landscape
Modalities
Routes of Administration
FGFR3 is amenable to small molecule drugs, with oral options available for convenient dosing.
The exclusive use of small molecules indicates a potential whitespace opportunity for alternative modalities like antibodies or biologics.
FGFR3 Clinical Trials 623 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on FGFR3. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 182 | 117 | 25 | 40 | 82% |
| Phase 2 | 347 | 129 | 70 | 145 | 65% |
| Phase 3 | 77 | 39 | 7 | 31 | 85% |
| Phase 4 | 17 | 11 | 1 | 5 | 92% |
Top Sponsors
By Modality
Top Conditions
Phase 3 Readout Calendar Pro
1 Phase 3 trial testing approved FGFR3 drugs across all sponsors.
Coverage: trials whose intervention is an approved drug targeting FGFR3. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.
FGFR3 Drug Approval Timeline (2009 - 2023)
The first FGFR3-targeting drug was approved in 2014, with the most recent approval in 2020.
The approval timeline indicates a relatively recent interest in FGFR3, suggesting continued development potential.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 8 companies competing
- • Market share by company
Full Drug Portfolio
- • All 8 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 8-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 235 (49 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 FGFR3. A trial counts if an approved drug acting on FGFR3 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.