Gilteritinib

Evidence Level: L5

Predicted Indications: 1

Gilteritinib

Pharmacist Assessment Report

Gilteritinib: From Acute Myeloid Leukaemia to Bulbar Polio

One-Sentence Summary

Gilteritinib is an oral kinase inhibitor originally used to treat FLT3-mutated acute myeloid leukaemia (AML). The TxGNN model (deep learning) predicts it may be effective for bulbar polio, but this is a model prediction only, with 0 clinical trials and 0 publications supporting it.

Quick Overview

Item	Content
Original Indication	Acute myeloid leukaemia (relapsed/refractory FLT3-mutated AML)
Predicted New Indication	Bulbar polio
TxGNN Prediction Score	0.9910
Evidence Level	L5
Australia Market Status	Listed on the PBS Schedule (1 of 1 items) – TGA (ARTG) registration status is not included in this data
Number of PBS Items	1 (valid: 1)
Recommended Decision	Hold

Why is This Prediction Reasonable?

Gilteritinib is a FLT3/AXL tyrosine kinase inhibitor. It is used in AML driven by FLT3 mutations. Detailed mechanism of action data was not available in the data compiled for this report, so this description relies on the drug’s known class.

No mechanistic link was identified between this mechanism and bulbar polio. Bulbar polio is a viral infection of motor neurons. Gilteritinib targets signalling pathways that drive leukaemic cell growth, and nothing in the data connects those pathways to poliovirus infection or motor neuron injury.

The high score (0.9910) comes from the TxGNN model (deep learning) and may reflect similarities the model learned between related diseases, not biological evidence. It should not be read as evidence of efficacy.

Clinical Trial Evidence

Currently no related clinical trials registered.

Literature Evidence

Currently no related literature available.

Australia Market Information

ARTG Number	Product Name	Dosage Form	Approved Indication
ARTG 13093D	Xospata	Tablet 40 mg (as fumarate)	Not stated in the data compiled for this report

The product is an oral, single-ingredient tablet and its registration status is valid.

Cytotoxicity

Item	Content
Cytotoxicity Classification	Targeted therapy (FLT3/AXL tyrosine kinase inhibitor)
Myelosuppression Risk	Please refer to the Product Information (PI) warnings and precautions
Emetogenicity Classification	Please refer to the Product Information (PI) warnings and precautions
Monitoring Items	Please refer to the Product Information (PI) warnings and precautions
Handling Protection	Please refer to the Product Information (PI) warnings and precautions

Safety Considerations

Drug Interactions: The interaction screen returned 100 interacting drugs, and 20 are shown in the data compiled for this report. Most are of moderate severity.
- Major: clarithromycin and dolasetron.
- Moderate (examples): aprepitant, cimetidine, famotidine, dexamethasone, levofloxacin, metformin, loperamide, palonosetron, naltrexone, miconazole, bisacodyl and linagliptin.
- Minor: metronidazole.

Please refer to the TGA-approved Product Information (PI) for warnings, contraindications and the full interaction list.

Conclusion and Next Steps

Decision: Hold

Rationale: The prediction rests only on a TxGNN model (deep learning) score, with no mechanistic link, trials or literature. Bulbar polio is a viral neurological disease with no biological connection to FLT3/AXL inhibition.

To proceed, the following is needed:

A plausible mechanistic hypothesis linking FLT3/AXL inhibition to poliovirus infection or motor neuron pathology
Preclinical evidence (for example, in vitro antiviral or neuroprotection data)
Detailed mechanism of action data and the TGA-approved Product Information, including warnings and contraindications
A review of whether a better-supported prediction should be assessed instead
Disclaimer

This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.