Ivacaftor
| Evidence Level: L5 | Predicted Indications: 50 |
Quick Overview
| Item | Value |
|---|---|
| Drug Name | Ivacaftor |
| DrugBank ID | DB08820 |
| Brand Names (EU) | Kalydeco |
| Evidence Level | L5 |
| Predicted Indications | 50 |
| Top Prediction Score | 96.97% |
Approved Indication (EMA)
Kalydeco tablets are indicated: - As monotherapy for the treatment of adults, adolescents, and children aged 6 years and older and weighing 25 kg or more with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating (Class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. - In a combination regimen with tezacaftor/ivacaftor tablets for the treatment
Predicted New Indications
TxGNN model predictions for potential drug repurposing:
| Rank | Indication | Score | Source |
|---|---|---|---|
| 1 | rheumatoid arthritis | 96.97% | DL |
| 2 | HIV infectious disease | 96.73% | DL |
| 3 | leprosy | 96.20% | DL |
| 4 | feline acquired immunodeficiency syndrome | 96.02% | DL |
| 5 | simian immunodeficiency virus infection | 96.02% | DL |
| 6 | multiple endocrine neoplasia | 95.82% | DL |
| 7 | neurodevelopmental disorder with ataxic gait, absent speech, and decreased cortical white matter | 95.54% | DL |
| 8 | brachydactyly-syndactyly syndrome | 95.52% | DL |
| 9 | colobomatous microphthalmia-rhizomelic dysplasia syndrome | 95.08% | DL |
| 10 | cytomegalovirus infection | 94.60% | DL |
| 11 | homozygous familial hypercholesterolemia | 94.49% | DL |
| 12 | infectious bovine rhinotracheitis | 94.18% | DL |
| 13 | malignant catarrh | 94.18% | DL |
| 14 | hereditary thrombocytopenia with normal platelets | 94.16% | DL |
| 15 | marcothrombocytopenia with mitral valve insufficiency | 94.16% | DL |
| 16 | transient neonatal thrombocytopenia | 94.07% | DL |
| 17 | thrombocytopenia | 94.04% | DL |
| 18 | gout | 93.95% | DL |
| 19 | systemic mastocytosis | 93.64% | DL |
| 20 | dense granule disease | 93.63% | 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.