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.
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 |
|---|---|---|---|
| NYVEPRIA | Pfizer | 2020 | 2 |
| STIMUFEND | Fresenius Kabi | 2022 | 2 |
| FULPHILA | Viatris | 2018 | 2 |
| RYZNEUTA | EVIVE BIOTECHNOLOGY | 2023 | 2 |
| ARMLUPEG | Lupin | 2025 | 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 148 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on CSF receptor. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 55 | 39 | 6 | 10 | 87% |
| Phase 2 | 65 | 34 | 12 | 18 | 74% |
| Phase 3 | 26 | 16 | 3 | 7 | 84% |
| 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
- • Sponsors: 90 (29 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 CSF receptor. A trial counts if an approved drug acting on CSF receptor 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.