Axitinib
| Evidence Level: L5 | Predicted Indications: 10 |
Table of Contents
Axitinib: Predicted Repurposing to Renal Cell Carcinoma Associated with Neuroblastoma
One-Sentence Summary
Axitinib is an oral VEGFR1-3 inhibitor marketed in the US as INLYTA, with a known role in renal cell carcinoma (RCC). The TxGNN model predicts it may be effective for renal cell carcinoma associated with neuroblastoma, a very rare entity. Currently 0 clinical trials and 0 publications support this specific prediction, so it rests on the model score alone.
Quick Overview
| Item | Content |
|---|---|
| Predicted New Indication | Renal cell carcinoma associated with neuroblastoma |
| TxGNN Prediction Score | 99.90% |
| Evidence Level | L5 |
| US Market Status | ✓ Marketed |
| Number of NDAs | 4 (all listed under NDA202324) |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Currently, detailed mechanism of action data is not available in the source record. Based on the available rationale, axitinib is a VEGFR1-3 inhibitor with established activity in renal cell carcinoma. Blocking VEGF signaling limits tumor angiogenesis, which is a central driver of many kidney cancers.
The predicted disease is also a renal cell carcinoma, so the biological link is plausible. However, no trial or publication addresses this rare neuroblastoma-associated entity. The prediction is therefore a model-based hypothesis, not a finding supported by clinical data.
Other axitinib predictions in the same output have stronger support. These include TFE3-translocation RCC (one small randomized Phase 2 trial) and collecting duct carcinoma (one ongoing Phase 2 trial). They are outside the scope of this report.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
Currently no related literature available.
US Market Information
| Authorization Number | Product Name | Dosage Form | Manufacturer |
|---|---|---|---|
| NDA202324 | INLYTA | Tablet, film coated (oral) | Pfizer Laboratories Div Pfizer Inc |
| NDA202324 | INLYTA | Tablet, film coated (oral) | U.S. Pharmaceuticals |
The source data lists four INLYTA entries, two per manufacturer, all under the same NDA number. No approved-indication text was provided.
Cytotoxicity
| Item | Content |
|---|---|
| Cytotoxicity Classification | Targeted therapy (VEGFR tyrosine kinase inhibitor) |
Please refer to the package insert warnings and precautions for myelosuppression risk, emetogenicity, monitoring items, and handling requirements.
Safety Considerations
Please refer to the package insert for safety information.
Conclusion and Next Steps
Decision: Hold
Rationale: This prediction has a very high model score but no supporting trials or publications (L5). The disease is extremely rare, so evidence is unlikely to accumulate soon. It should not advance beyond the model-prediction stage without at least some clinical or case-level data.
To proceed, the following is needed:
- Any case reports, registries, or trials for neuroblastoma-associated RCC.
- Molecular characterization of this entity to test whether it is VEGF-dependent.
- Detailed mechanism of action data (MOA).
- The US package insert (warnings, contraindications, approved indications) for safety screening.
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.