Glycerol Phenylbutyrate
| Evidence Level: L5 | Predicted Indications: 50 |
Quick Overview
| Item | Value |
|---|---|
| Drug Name | Glycerol Phenylbutyrate |
| DrugBank ID | DB08909 |
| Brand Names (EU) | Ravicti |
| Evidence Level | L5 |
| Predicted Indications | 50 |
| Top Prediction Score | 98.67% |
Approved Indication (EMA)
Ravicti is indicated for use as adjunctive therapy for chronic management of patients with urea cycle disorders (UCDs) including deficiencies of carbamoyl phosphate-synthase-I (CPS), ornithine carbamoyltransferase (OTC), argininosuccinate synthetase (ASS), argininosuccinate lyase (ASL), arginase I (ARG) and ornithine translocase deficiency hyperornithinaemia-hyperammonaemia homocitrullinuria syndrome (HHH) who cannot be managed by dietary protein restriction and/or amino acid supplementatio
Predicted New Indications
TxGNN model predictions for potential drug repurposing:
| Rank | Indication | Score | Source |
|---|---|---|---|
| 1 | urea cycle disorder | 98.67% | DL |
| 2 | argininosuccinic aciduria | 98.25% | DL |
| 3 | carbamoyl phosphate synthetase I deficiency disease | 97.92% | DL |
| 4 | acute neonatal citrullinemia type I | 97.24% | DL |
| 5 | adult-onset citrullinemia type I | 97.24% | DL |
| 6 | citrullinemia, type II, adult-onset | 97.13% | DL |
| 7 | 3-methylcrotonyl-CoA carboxylase 1 deficiency | 97.10% | DL |
| 8 | hyperinsulinism-hyperammonemia syndrome | 96.78% | DL |
| 9 | hyperammonemic encephalopathy due to carbonic anhydrase VA deficiency | 96.56% | DL |
| 10 | hyperargininemia | 94.98% | DL |
| 11 | citrullinemia | 94.31% | DL |
| 12 | citrin deficiency | 93.45% | DL |
| 13 | neonatal intrahepatic cholestasis due to citrin deficiency | 92.72% | DL |
| 14 | benign neoplasm of adrenal gland | 90.80% | DL |
| 15 | hyperammonemia due to N-acetylglutamate synthase deficiency | 89.01% | DL |
| 16 | familial apolipoprotein C-II deficiency | 86.32% | DL |
| 17 | polycystic kidney disease 3 with or without polycystic liver disease | 83.08% | DL |
| 18 | acute intermittent porphyria | 81.32% | DL |
| 19 | renal-hepatic-pancreatic dysplasia | 79.64% | DL |
| 20 | autosomal ichthyosis syndrome with fatal disease course | 79.47% | 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.