Miltefosine

Evidence Level: L5

Predicted Indications: 3

Miltefosine

Pharmacist Assessment Report

Miltefosine: From Leishmaniasis to echinococcus granulosus infectious disease

One-Sentence Summary

Miltefosine is an oral drug with known uses in leishmaniasis, including mucocutaneous and visceral forms. The TxGNN model (deep learning) predicts it may be useful for echinococcus granulosus infectious disease, but no clinical trials and no publications directly support this prediction, so it rests on the model score alone. This score comes from the TxGNN model (deep learning) and may reflect similarities the model learned between related diseases, not biological evidence.

Quick Overview

Item	Content
Original Indication	Leishmaniasis, mucocutaneous leishmaniasis, visceral leishmaniasis (relations recorded in the knowledge graph; the India licence text contains no indication wording)
Predicted New Indication	Echinococcus granulosus infectious disease
TxGNN Prediction Score	0.9941
Evidence Level	L5
India Market Status	Listed in a compiled Indian medicine price dataset (1 of 1) – CDSCO approval status is not included in this data
Number of Listed Products	1 (valid: 1)
Recommended Decision	Hold

Why is This Prediction Reasonable?

Currently, detailed mechanism of action data is not available for this report. Based on general knowledge, miltefosine is an alkylphosphocholine compound with activity against Leishmania parasites. Its effects are thought to involve disruption of lipid metabolism and membrane signalling. The data compiled for this report also lists AKT1 as a pharmacological target.

Leishmaniasis is a protozoan infection, while Echinococcus granulosus is a tapeworm (cestode) that causes cystic echinococcosis. Both are parasitic diseases, so a shared vulnerability in parasite membrane or lipid signalling is conceivable. Whether miltefosine acts on Echinococcus granulosus has not been verified in the data compiled here. This is a hypothesis generated by the model, not a demonstrated link.

Two other predictions were also generated: alveolar echinococcosis (score 0.9921) and smouldering systemic mastocytosis (score 0.9905). Both likewise have no direct supporting studies of miltefosine. The only publication retrieved for alveolar echinococcosis tested other drugs (itraconazole, methiazole, nitazoxanide) in vitro and does not evaluate miltefosine. The link to mastocytosis is biologically unclear.

Clinical Trial Evidence

Currently no related clinical trials registered.

Literature Evidence

Currently no related literature available for Echinococcus granulosus infection.

India Market Information

Authorization Number	Product Name	Dosage Form	Approved Indication
Registration No. 108465	Impavido 50mg Capsule (Zydus Cadila)	Strip of 7 capsules (oral)	Not stated in the licence record

This is one valid single-ingredient licence. The approved indication text is empty in the data compiled, so the licence cannot be used to confirm any specific approved use.

Cytotoxicity

Not displayed: miltefosine is not classified as an antineoplastic drug in the data compiled here, and its recorded indications are parasitic infections.

Safety Considerations

Drug Interactions: Ten interaction records were found. Nine are rated Moderate and relate to hormonal agents: levonorgestrel, norethisterone, desogestrel, ethinylestradiol, drospirenone, dienogest, norgestrel and norgestimate (hormonal contraceptive components), plus idelalisib. The tenth is a pharmacology record of AKT1 as a target of miltefosine, which is a target annotation rather than a clinical interaction.
Knowledge-graph contraindication relations (not label text): Sjogren-Larsson syndrome and congenital ichthyosiform erythroderma.

Please refer to the package insert for warnings and contraindications, as none were available in the data compiled.

Conclusion and Next Steps

Decision: Hold

Rationale: The prediction for Echinococcus granulosus infection rests only on a high model score. No trial or publication directly studies miltefosine in this disease, so the evidence level is L5. Warnings and contraindications from the label are also missing.

To proceed, the following is needed:

Preclinical data (in vitro and animal) on miltefosine against Echinococcus granulosus and E. multilocularis
Mechanism of action data for miltefosine
The India package insert, covering warnings, contraindications and approved indication text
A safety review of the hormonal contraceptive interactions and the knowledge-graph contraindication relations
A comparison against standard therapy (e.g., albendazole) before any clinical study design
Disclaimer

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