Elexacaftor
| Evidence Level: L5 | Predicted Indications: 50 |
Quick Overview
| Item | Value |
|---|---|
| Drug Name | Elexacaftor |
| DrugBank ID | DB15444 |
| Brand Names (EU) | Elexacaftor |
| Evidence Level | L5 |
| Predicted Indications | 50 |
| Top Prediction Score | 98.11% |
Approved Indication (EMA)
Kaftrio tablets are indicated in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one non-Class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Kaftrio granules are indicated in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in paediatric patients aged 2 to less than 6 years who have at least one non-Class I mutation in the cystic fibrosis transmem
Predicted New Indications
TxGNN model predictions for potential drug repurposing:
| Rank | Indication | Score | Source |
|---|---|---|---|
| 1 | rheumatoid arthritis | 98.11% | DL |
| 2 | amyotrophic lateral sclerosis | 98.00% | DL |
| 3 | leprosy | 98.00% | DL |
| 4 | multiple endocrine neoplasia | 97.85% | DL |
| 5 | Mills syndrome | 97.62% | DL |
| 6 | amyotrophic lateral sclerosis, susceptibility to | 97.62% | DL |
| 7 | amyotrohpic lateral sclerosis type 22 | 97.60% | DL |
| 8 | migraine with or without aura, susceptibility to | 97.55% | DL |
| 9 | nephrogenic syndrome of inappropriate antidiuresis | 97.55% | DL |
| 10 | pulmonary hypertension | 97.45% | DL |
| 11 | axial spondylometaphyseal dysplasia | 97.43% | DL |
| 12 | brachydactyly-syndactyly syndrome | 97.33% | DL |
| 13 | bilateral parasagittal parieto-occipital polymicrogyria | 97.31% | DL |
| 14 | migraine disorder | 97.28% | DL |
| 15 | kyphoscoliotic heart disease | 97.22% | DL |
| 16 | homozygous familial hypercholesterolemia | 97.20% | DL |
| 17 | trichomegaly-retina pigmentary degeneration-dwarfism syndrome | 97.11% | DL |
| 18 | lethal arthrogryposis-anterior horn cell disease syndrome | 97.09% | DL |
| 19 | female breast carcinoma | 97.01% | DL |
| 20 | monomelic amyotrophy | 96.98% | 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.