PDGFR-alpha Inhibitors
9 drugsAbout PDGFR-alpha
PDGFR-alpha is a receptor tyrosine kinase crucial for cell growth, differentiation, and survival. Activated by PDGFs, it initiates intracellular signaling cascades that regulate fundamental cellular processes.
PDGFR-alpha is a therapeutic target because of its role in cell proliferation, though there is currently no genetic evidence directly linking it to specific diseases.
PDGFR-alpha is targeted by one FDA-approved small molecule drug, STIVARGA (Bayer), which has three indications.
PDGFR-alpha Genetic Evidence Strong
There is no genetic evidence data available for PDGFR-alpha.
Lack of genetic support suggests higher risk in drug development, warranting careful patient stratification strategies.
💡 Why inhibition?
- • Loss-of-function variants reduce disease risk (OR < 1)
- • 100% directional consistency across 1 traits
- • Strong signal in endocrine system disease, reproductive system or breast disease, cancer or benign tumor pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
13 totalGWAS and other genetic studies link PDGFRA to 13 diseases.
🔗 Colocalization Evidence 20 strong
max H4: 1.00eQTL/pQTL signals for PDGFRA 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 PDGFR-alpha Drugs
Bayer is the only company with an approved drug targeting PDGFR-alpha.
The market is highly concentrated, indicating potential entry barriers or limited investment in this target.
| Drug | Company | Approved | Indications |
|---|---|---|---|
| RYDAPT | Novartis | 2017 | 4 |
| STIVARGA | Bayer | 2012 | 3 |
| SUNITINIB MALATE | Dr. Reddy's | 2021 | 3 |
| VOTRIENT | Novartis | 2009 | 2 |
| PAZOPANIB HYDROCHLORIDE | EUGIA PHARMA | 2023 | 2 |
| QINLOCK | DECIPHERA PHARMS | 2020 | 1 |
PDGFR-alpha Drug Modality Landscape
Modalities
Routes of Administration
PDGFR-alpha is amenable to small molecule drugs, with oral options available for convenient dosing.
Exploring alternative modalities like antibodies or PROTACs could provide differentiation and competitive advantage.
PDGFR-alpha Clinical Trials 903 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on PDGFR-alpha. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 243 | 146 | 42 | 54 | 78% |
| Phase 2 | 529 | 228 | 114 | 184 | 67% |
| Phase 3 | 112 | 54 | 13 | 45 | 81% |
| Phase 4 | 19 | 14 | 2 | 3 | 88% |
Top Sponsors
By Modality
Top Conditions
Phase 3 Readout Calendar Pro
5 Phase 3 trials testing approved PDGFR-alpha drugs across all sponsors.
Coverage: trials whose intervention is an approved drug targeting PDGFR-alpha. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.
PDGFR-alpha Drug Approval Timeline (2006 - 2023)
The first and only drug, STIVARGA, was approved in 2012.
The approval timeline shows no recent activity, suggesting a saturated market or challenges in developing new PDGFR-alpha inhibitors.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 8 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: 357 (76 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 PDGFR-alpha. A trial counts if an approved drug acting on PDGFR-alpha 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.