Lumacaftor
| Evidence Level: L5 | Predicted Indications: 50 |
Quick Overview
| Item | Value |
|---|---|
| Drug Name | Lumacaftor |
| DrugBank ID | DB09280 |
| Brand Names (EU) | Lumacaftor |
| Evidence Level | L5 |
| Predicted Indications | 50 |
| Top Prediction Score | 99.44% |
Approved Indication (EMA)
Orkambi tablets are indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who are homozygous for the F508del mutation in the CFTR gene. Orkambi granules are indicated for the treatment of cystic fibrosis (CF) in children aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene.
Predicted New Indications
TxGNN model predictions for potential drug repurposing:
| Rank | Indication | Score | Source |
|---|---|---|---|
| 1 | leprosy | 99.44% | DL |
| 2 | migraine with or without aura, susceptibility to | 98.85% | DL |
| 3 | migraine disorder | 98.73% | DL |
| 4 | rheumatoid arthritis | 98.69% | DL |
| 5 | pulmonary hypertension | 98.67% | DL |
| 6 | kyphoscoliotic heart disease | 98.52% | DL |
| 7 | migraine with brainstem aura | 98.51% | DL |
| 8 | multiple endocrine neoplasia | 98.36% | DL |
| 9 | brachydactyly-syndactyly syndrome | 98.17% | DL |
| 10 | nephrogenic syndrome of inappropriate antidiuresis | 98.00% | DL |
| 11 | homozygous familial hypercholesterolemia | 97.95% | DL |
| 12 | Prinzmetal angina | 97.91% | DL |
| 13 | colobomatous microphthalmia-rhizomelic dysplasia syndrome | 97.84% | DL |
| 14 | atrophoderma vermiculata | 97.50% | DL |
| 15 | ulerythema ophryogenesis | 97.12% | DL |
| 16 | amyotrophic lateral sclerosis | 96.88% | DL |
| 17 | persistent Mullerian duct syndrome | 96.65% | DL |
| 18 | coronary artery disease | 96.56% | DL |
| 19 | pneumocystosis | 96.50% | DL |
| 20 | hypoalphalipoproteinemia | 96.50% | 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.