KIT Inhibitors
23 drugsAbout KIT
KIT (KIT Proto-Oncogene Receptor Tyrosine Kinase) is a receptor tyrosine kinase on the cell surface involved in cell signaling pathways that regulate cell growth, differentiation, and survival. Activated KIT triggers intracellular events that can lead to uncontrolled cell proliferation.
Human genetics provide strong validation for KIT as a therapeutic target (max score 0.92), with variants linked to cutaneous mastocytosis, piebaldism and gastrointestinal stromal tumor. Loss-of-function variants are associated with increased risk for cutaneous mastocytosis and piebaldism, while also protecting against gastrointestinal stromal tumor, suggesting context-dependent therapeutic strategies.
KIT is targeted by 16 FDA-approved small molecule drugs including AYVAKIT, LENVIMA and CABOMETYX. While most approved drugs are used in other therapeutic areas, 4 are used in oncology, highlighting KIT's relevance in cancer treatment.
KIT Genetic Evidence Strong
KIT has strong genetic support with a maximum score of 0.92 across 18 diseases.
Strong genetic support suggests a higher likelihood of clinical success, potentially justifying investment in novel therapies.
💡 Why activation?
- • Loss-of-function variants increase disease risk (OR > 1) — restoring function may help
- • 67% directional consistency across 3 traits
- • Strong signal in hematologic disease, immune system disease, integumentary system disease pathways
Cross-Disease Effects
Trade-off: ModerateDirection of Effect
67% alignedEvidence Across Diseases
18 totalGWAS and other genetic studies link KIT to 18 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 KIT 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 KIT Drugs
Fourteen companies have approved drugs targeting KIT, with Viatris, DECIPHERA PHARMS and NOVUGEN among the top players.
The competitive landscape suggests moderate market fragmentation, potentially lowering entry barriers for new companies.
| Drug | Company | Approved | Indications |
|---|---|---|---|
| LENVIMA | EISAI INC | 2015 | 5 |
| SUTENT | CPPI CV | 2006 | 4 |
| RYDAPT | Novartis | 2017 | 4 |
| STIVARGA | Bayer | 2012 | 3 |
| SUNITINIB MALATE | Dr. Reddy's | 2021 | 3 |
| SORAFENIB TOSYLATE | YABAO PHARM | 2020 | 3 |
| NEXAVAR | Bayer | 2005 | 3 |
| DASATINIB | Teva | 2016 | 2 |
| VOTRIENT | Novartis | 2009 | 2 |
| PAZOPANIB HYDROCHLORIDE | EUGIA PHARMA | 2023 | 2 |
| SPRYCEL | Bristol-Myers Squibb | 2006 | 2 |
| ICLUSIG | Takeda | 2012 | 2 |
| PHYRAGO | HANDA THERAP | 2023 | 2 |
| FOTIVDA | AVEO PHARMS | 2021 | 1 |
| QINLOCK | DECIPHERA PHARMS | 2020 | 1 |
| TURALIO | DAIICHI SANKYO INC | 2019 | 1 |
| TASIGNA | Novartis | 2007 | 1 |
| DANZITEN | AZURITY | 2024 | 1 |
| COMETRIQ | EXELIXIS | 2012 | 1 |
| RESNIBEN | AZURITY | - | - |
KIT Drug Modality Landscape
Modalities
Routes of Administration
KIT is amenable to small molecule drugs, with oral options available for convenient dosing.
The absence of other modalities like antibodies or fusion proteins represents a whitespace opportunity for innovation.
KIT Clinical Trials 1,972 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on KIT. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 617 | 374 | 129 | 112 | 74% |
| Phase 2 | 1057 | 501 | 239 | 311 | 68% |
| Phase 3 | 245 | 122 | 41 | 82 | 75% |
| Phase 4 | 53 | 36 | 6 | 11 | 86% |
Top Sponsors
By Modality
Top Conditions
Phase 3 Readout Calendar Pro
8 Phase 3 trials testing approved KIT drugs across all sponsors.
Coverage: trials whose intervention is an approved drug targeting KIT. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.
KIT Drug Approval Timeline (2005 - 2024)
The first KIT-targeting drug was approved in 2005, with the most recent approval in 2023, spanning 19 years.
The continued approval of new drugs indicates sustained interest and potential for further development in this target space.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 18 companies competing
- • Market share by company
Full Drug Portfolio
- • All 23 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 23-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 625 (138 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 KIT. A trial counts if an approved drug acting on KIT 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.