Nifurtimox
| Evidence Level: L5 | Predicted Indications: 7 |
Table of Contents
Nifurtimox: From Antiparasitic Therapy to Congenital Analbuminemia
One-Sentence Summary
Nifurtimox is a nitrofuran antiparasitic drug, marketed in the US as LAMPIT film-coated tablets. The TxGNN model predicts it may be effective for congenital analbuminemia, but 0 clinical trials and 0 publications currently support this prediction, so it rests on the model score alone.
Quick Overview
| Item | Content |
|---|---|
| Predicted New Indication | Congenital analbuminemia |
| TxGNN Prediction Score | 99.58% |
| Evidence Level | L5 |
| US Market Status | ✓ Marketed |
| Number of NDAs | 2 records (both NDA213464) |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Currently, detailed mechanism of action data is not available in the source data. Based on known information, nifurtimox is a nitrofuran antiparasitic. Its activity is thought to come from nitroreductase-mediated generation of reactive oxygen species and nitro-radical metabolites in trypanosomes.
No plausible mechanistic link to congenital analbuminemia was identified. This is a rare genetic disorder of albumin synthesis, and a drug that acts on parasite nitroreductase pathways has no evident way to correct it. The high score (0.996) is a graph-based prediction with no supporting biological rationale.
The other six predictions show the same pattern. All are L5, with scores of 0.991–0.995 and no trials or literature:
- polyclonal hyperviscosity syndrome
- hyperamylasemia
- blood group incompatibility
- monoclonal gammopathy
- premalignant hematological system disease
- hematological disease associated with an acquired peripheral neuropathy
The first two share the same score (0.9947), which suggests a shared graph-neighborhood artifact rather than disease-specific signal. Peripheral neuropathy is a known adverse effect of nifurtimox, so for the last of these the drug could worsen the condition rather than treat it.
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 |
|---|---|---|
| NDA213464 | LAMPIT (Bayer Healthcare Pharmaceuticals Inc.) | Film-coated tablet (oral) |
The source data lists NDA213464 twice with identical content, and no approved-indication text is included.
Safety Considerations
- Adverse effects: Peripheral neuropathy is a known adverse effect of nifurtimox. This matters especially for the neuropathy-related prediction (rank 7).
- Drug Interactions: No interaction records were found in the queried source.
Please refer to the package insert for other safety information (warnings and contraindications).
Conclusion and Next Steps
Decision: Hold
Rationale: The prediction is model-only (L5, no trials or literature), and no plausible mechanistic link to congenital analbuminemia exists. Safety data are also incomplete, with the package insert warnings still missing.
To proceed, the following is needed:
- FDA package insert warnings and contraindications (blocking gap for safety screening)
- Mechanism of action data from DrugBank
- A biological rationale connecting nitrofuran activity to albumin synthesis, or evidence from preclinical studies
- A safety assessment of the peripheral neuropathy risk before evaluating any neuropathy-related indication
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.