G-CSF receptor Inhibitors
7 drugsAbout G-CSF receptor
The Granulocyte Colony-Stimulating Factor Receptor (G-CSF receptor) stimulates the production, differentiation, and activation of neutrophils, which are essential for fighting infection. As a key regulator of the immune system, it represents a compelling target for therapeutic intervention.
G-CSF receptor is strongly supported by genetics (max score 0.93), with variants linked to severe congenital neutropenia (score 0.93), autosomal recessive severe congenital neutropenia (score 0.84), and hereditary neutrophilia (score 0.74). Loss-of-function variants are associated with increased disease risk, suggesting activation may be beneficial.
Five FDA-approved biologic drugs target G-CSF receptor, including ZARXIO, RELEUKO, NYPOZI, NIVESTYM, and NEUPOGEN. These drugs are all approved for oncology indications, particularly managing chemotherapy-induced neutropenia.
G-CSF receptor Genetic Evidence Strong
G-CSF receptor has strong genetic support with a maximum score of 0.93 across 11 diseases.
The strong genetic support suggests that clinical trials targeting G-CSF receptor have a higher likelihood of 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, hematologic disease, immune system disease pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
11 totalGWAS and other genetic studies link CSF3R to 11 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 CSF3R 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 G-CSF receptor Drugs
Five companies have approved drugs targeting G-CSF receptor, including Novartis, KASHIV BIOSCIENCES LLC, TANVEX BIOPHARMA USA INC, Pfizer, and Amgen.
The presence of multiple companies suggests a competitive market with moderate barriers to entry.
| Drug | Company | Approved | Indications |
|---|---|---|---|
| RELEUKO | KASHIV BIOSCIENCES LLC | 2022 | 7 |
| NIVESTYM | Pfizer | 2018 | 6 |
| GRANIX | SICOR BIOTECH | 2012 | 3 |
| ROLVEDON | SPECTRUM PHARMS | 2022 | 2 |
G-CSF receptor Drug Modality Landscape
Modalities
Routes of Administration
G-CSF receptor requires biologic approaches (biologic (other)), likely due to its structure or location.
The lack of small molecule or antibody drugs indicates a potential whitespace opportunity for novel modalities.
G-CSF receptor Clinical Trials 250 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on G-CSF receptor. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 108 | 46 | 29 | 33 | 61% |
| Phase 2 | 99 | 56 | 16 | 26 | 78% |
| Phase 3 | 33 | 16 | 10 | 7 | 62% |
| Phase 4 | 10 | 5 | 3 | 2 | 63% |
Top Sponsors
By Modality
Top Conditions
G-CSF receptor Drug Approval Timeline (1991 - 2024)
The first drug was approved in 1991 (NEUPOGEN), and the most recent in 2024 (NYPOZI), spanning 34 years.
The continued approvals indicate sustained interest and potential for further innovation in targeting G-CSF receptor.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 7 companies competing
- • Market share by company
Full Drug Portfolio
- • All 7 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 7-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 117 (32 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 G-CSF receptor. A trial counts if an approved drug acting on G-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.