Onasemnogene Abeparvovec
| Evidence Level: L5 | Predicted Indications: 50 |
Quick Overview
| Item | Value |
|---|---|
| Drug Name | Onasemnogene Abeparvovec |
| DrugBank ID | DB15528 |
| Brand Names (EU) | Zolgensma |
| Evidence Level | L5 |
| Predicted Indications | 50 |
| Top Prediction Score | 86.13% |
Approved Indication (EMA)
Zolgensma is indicated for the treatment of: patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.
Predicted New Indications
TxGNN model predictions for potential drug repurposing:
| Rank | Indication | Score | Source |
|---|---|---|---|
| 1 | bronchitis | 86.13% | DL |
| 2 | severe nonproliferative diabetic retinopathy | 79.85% | DL |
| 3 | diabetic retinopathy | 77.44% | DL |
| 4 | bronchial neoplasm (disease) | 77.10% | DL |
| 5 | non-seminomatous lesion | 76.78% | DL |
| 6 | chondroid hamartoma | 76.78% | DL |
| 7 | ductal or ductular proliferation | 76.78% | DL |
| 8 | bronchial adenomas/carcinoids childhood | 76.78% | DL |
| 9 | rectosigmoid junction neoplasm | 76.70% | DL |
| 10 | tumor of testis and paratestis | 76.67% | DL |
| 11 | colonic lymphangioma | 76.59% | DL |
| 12 | lipoma of colon | 76.51% | DL |
| 13 | odontogenic cyst | 76.46% | DL |
| 14 | cecum neuroendocrine tumor G1 | 76.44% | DL |
| 15 | thyroglossal duct cyst | 76.43% | DL |
| 16 | colonic neoplasm | 76.42% | DL |
| 17 | cecal disease | 76.39% | DL |
| 18 | cecum villous adenoma | 76.38% | DL |
| 19 | cystic neoplasm | 76.35% | DL |
| 20 | cavernous hemangioma of colon | 76.32% | DL |
Showing top 20 of 50 predictions.
About TxGNN Predictions
Prediction Sources
| Source | Description |
|---|---|
| KG | Knowledge Graph - Network topology-based associations |
| DL | Deep Learning - Neural network score prediction |
Evidence Levels
| Level | Definition |
|---|---|
| L1 | Multiple Phase 3 RCTs / Systematic Reviews |
| L2 | Single RCT or multiple Phase 2 trials |
| L3 | Observational studies / Large case series |
| L4 | Preclinical / Mechanistic / Case reports |
| L5 | AI prediction only (current) |
Clinical Validation Needed
Research Use Only: These predictions are computational hypotheses that require clinical validation. They should NOT be used for clinical decision-making.
Next Steps for Validation
- Literature Review: Search PubMed for existing evidence
- Clinical Trial Search: Check ClinicalTrials.gov for ongoing studies
- Mechanistic Analysis: Evaluate biological plausibility
- Preclinical Studies: Conduct in vitro/in vivo validation
- Clinical Trials: Design and conduct human studies
Data Access
- FHIR API:
/fhir/ClinicalUseDefinition/ - CSV Download: All Predictions
- GitHub: yao-care/EuTxGNN
Citation
If using this data, please cite:
@article{huang2023txgnn,
title={A foundation model for clinician-centered drug repurposing},
author={Huang, Kexin and others},
journal={Nature Medicine},
year={2023},
doi={10.1038/s41591-023-02233-x}
}
Disclaimer: This report is for research purposes only and does not constitute medical advice. Drug repurposing predictions require rigorous clinical validation before any therapeutic application.