Tezacaftor

Evidence Level: L5 Predicted Indications: 50

Quick Overview

Item Value
Drug Name Tezacaftor
DrugBank ID DB11712
Brand Names (EU) Tezacaftor
Evidence Level L5
Predicted Indications 50
Top Prediction Score 99.24%

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 HIV infectious disease 99.24% DL
2 leprosy 99.14% DL
3 multiple endocrine neoplasia 99.06% DL
4 simian immunodeficiency virus infection 98.96% DL
5 feline acquired immunodeficiency syndrome 98.96% DL
6 neurodevelopmental disorder with ataxic gait, absent speech, and decreased cortical white matter 98.85% DL
7 rheumatoid arthritis 98.65% DL
8 female breast carcinoma 98.39% DL
9 homozygous familial hypercholesterolemia 98.37% DL
10 amyotrophic lateral sclerosis 98.35% DL
11 thrombocytopenia 98.35% DL
12 hereditary thrombocytopenia with normal platelets 98.08% DL
13 marcothrombocytopenia with mitral valve insufficiency 98.07% DL
14 brachydactyly-syndactyly syndrome 98.05% DL
15 transient neonatal thrombocytopenia 98.02% DL
16 oral candidiasis 97.92% DL
17 collagenopathy 97.90% DL
18 dense granule disease 97.88% DL
19 Mills syndrome 97.88% DL
20 amyotrohpic lateral sclerosis type 22 97.87% 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

  1. Literature Review: Search PubMed for existing evidence
  2. Clinical Trial Search: Check ClinicalTrials.gov for ongoing studies
  3. Mechanistic Analysis: Evaluate biological plausibility
  4. Preclinical Studies: Conduct in vitro/in vivo validation
  5. Clinical Trials: Design and conduct human studies

Data Access


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.

Copyright © 2026 EuTxGNN Project. For research purposes only. Not medical advice.