Fostamatinib
| Evidence Level: L5 | Predicted Indications: 2 |
Table of Contents
- Fostamatinib
- Fostamatinib: From Immune Thrombocytopenia to Autosomal Thrombocytopenia with Normal Platelets
Fostamatinib: From Immune Thrombocytopenia to Autosomal Thrombocytopenia with Normal Platelets
One-Sentence Summary
Fostamatinib is marketed in the US as TAVALISSE for immune thrombocytopenia (from general drug knowledge; the supplied data lists no indication text). The TxGNN model predicts it may be useful for autosomal thrombocytopenia with normal platelets, but no clinical trials and no publications currently support this prediction.
Quick Overview
| Item | Content |
|---|---|
| Predicted New Indication | Autosomal thrombocytopenia with normal platelets |
| TxGNN Prediction Score | 99.45% |
| Evidence Level | L5 (model prediction only) |
| US Market Status | ✓ Marketed |
| Number of NDAs | 2 listed entries, both under NDA209299 |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Detailed mechanism of action data is not available in the supplied input. From general drug knowledge, fostamatinib is a SYK inhibitor whose active metabolite is R406. In immune thrombocytopenia it reduces Fc-gamma-receptor-mediated platelet destruction by macrophages.
The predicted disease is an inherited thrombocytopenia, usually linked to ANKRD26 variants that dysregulate thrombopoietin-receptor signalling and impair platelet production. Its cause is not immune clearance, so the SYK-inhibition mechanism does not obviously apply. The link appears to be a weak, indirect inference from the shared low-platelet phenotype in the knowledge graph.
A high model score is not evidence of efficacy. The prediction needs independent mechanistic and clinical support before it can be considered credible.
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 |
|---|---|---|---|
| NDA209299 | TAVALISSE | Tablet (oral) | Rigel Pharmaceuticals, Inc. |
The input lists this NDA twice with identical details, so it is shown once here.
Safety Considerations
Please refer to the package insert for safety information.
Conclusion and Next Steps
Decision: Hold
Rationale: The only support is a model score, with no trials or publications. The SYK-inhibition mechanism does not fit an inherited platelet-production disorder. The second-ranked prediction, non-syndromic esophageal malformation (score 99.05%), has no plausible mechanism for a SYK inhibitor and is likely a knowledge-graph artifact.
To proceed, the following is needed:
- Package insert warnings and contraindications (a blocking gap for safety screening)
- Mechanism of action data from DrugBank
- Preclinical or mechanistic evidence that SYK inhibition affects platelet production in ANKRD26-related thrombocytopenia
- Any registered trials or published case reports in this indication
This report is for research reference only and does not constitute medical advice. Repurposing candidates require clinical validation before any use.
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.