CSF receptor Inhibitors
9 drugsAbout CSF receptor
CSF1R is a receptor involved in various biological processes. Genetic studies show strong evidence linking CSF1R to diseases like hereditary diffuse leukoencephalopathy (score 0.93). Loss-of-function variants increase disease risk.
Human genetics strongly support CSF1R as a therapeutic target. Activation of CSF1R is likely beneficial based on genetic data. This suggests agonist therapies could be effective.
Nine FDA-approved drugs target CSF1R, all biologics. These include RYZNEUTA, FYLNETRA, NEULASTA, UDENYCA, and ZIEXTENZO. The first drug was approved in 2002, with the most recent in 2025.
Strategic Insights
ℹ️ How we calculate- White space opportunity in Recurrent Chronic Myelomonocytic Leukemia with only 3 trials.
CSF receptor Genetic Evidence Strong
Strong genetic evidence supports CSF1R's role in diseases like leukoencephalopathy.
High genetic scores (up to 0.93) suggest 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, nervous system disease pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
7 totalGWAS and other genetic studies link CSF1R to 7 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 CSF1R 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 CSF receptor Drugs
Nine companies have approved drugs targeting CSF1R.
Relatively low market concentration suggests moderate entry barriers.
| Drug | Company | Approved | Indications |
|---|---|---|---|
| STIMUFEND | Fresenius Kabi | 2022 | 2 |
| ARMLUPEG | Lupin | 2025 | 2 |
| NEULASTA | Amgen | 2002 | 2 |
| UDENYCA | COHERUS BIOSCIENCES INC | 2018 | 2 |
| RYZNEUTA | EVIVE BIOTECHNOLOGY | 2023 | 2 |
| FYLNETRA | KASHIV BIOSCIENCES LLC | 2022 | 2 |
CSF receptor Drug Modality Landscape
Modalities
Routes of Administration
CSF receptor requires biologic approaches (biologic (other)), likely due to its structure or location.
The absence of small molecule drugs indicates a whitespace opportunity.
📈 Modality Evolution
other biologics pioneered CSF receptor targeting (2002), with enzymes entering more recently (2023).
CSF receptor Clinical Trials 146 trials
Completion by Phase
| Phase | Total | Completed | Failed | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 54 | 39 | 6 | 9 | 87% |
| Phase 2 | 65 | 34 | 12 | 19 | 74% |
| Phase 3 | 25 | 15 | 3 | 7 | 83% |
| Phase 4 | 2 | 2 | 0 | 0 | 100% |
Top Sponsors
By Modality
CSF receptor Drug Approval Timeline (2002 - 2025)
Approvals span 24 years, from 2002 to 2025.
Recent approval (2025) indicates continued interest in this target.
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
- • Competition: High (15 sponsors)
- • White space: 10 underexplored indications
- • Success rates by condition
Full summary • All drugs • Genetic evidence • Trials • Timeline
How We Calculate These Metrics
Target Attractiveness Score
A 0-100 score based on trial activity, sponsor diversity, and completion rates. Calculated from 164 clinical trials targeting CSF receptor.
Completion rate: Percentage of trials that reached their planned endpoint. Trials terminated early, withdrawn, or suspended are not counted—these often indicate safety issues, lack of efficacy, or strategic pivots.
- Highly Attractive (80+): High trial activity, many sponsors, strong completion rates
- Attractive (60-79): Good trial activity and validation
- Moderate (40-59): Moderate interest from sponsors
- Low (under 40): Limited trial activity or validation concerns
Strategic Insights
Auto-generated insights based on trial analytics including competition intensity, white space opportunities, modality shifts, and failure patterns. We analyze trial sponsors, phases, indications, and outcomes.
Risk Signals
- High Competition: Many sponsors competing for this target (may reduce market opportunity)
- High Failure Risk: Low trial completion rates suggest development challenges
- Low Validation: Limited trial activity or poor outcomes indicate uncertain viability
- White Space Available: Underexplored indications present opportunities