Eliglustat (Tartrate)

Evidence Level: L5 Predicted Indications: 50

Quick Overview

Item Value
Drug Name Eliglustat (Tartrate)
DrugBank ID DB09039
Brand Names (EU) Cerdelga
Evidence Level L5
Predicted Indications 50
Top Prediction Score 98.51%

Approved Indication (EMA)

AdultsCerdelga is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1), who are CYP2D6 poor metabolisers (PMs), intermediate metabolisers (IMs) or extensive metabolisers (EMs).Paediatric population (from 6 to < 18 years of age) weighing ≥ 15 kgCerdelga is indicated for paediatric patients with GD1 who are 6 years and older with a minimum body weight of 15 kg, who are stable on enzyme replacement therapy (ERT), and who are CYP2D6 PMs, IMs or EMs. 

Predicted New Indications

TxGNN model predictions for potential drug repurposing:

Rank Indication Score Source
1 Gaucher disease 98.51% DL
2 autosomal ichthyosis syndrome with fatal disease course 98.42% DL
3 benign neoplasm of adrenal gland 98.33% DL
4 polycystic kidney disease 3 with or without polycystic liver disease 96.91% DL
5 gastrocutaneous syndrome 96.67% DL
6 familial generalized lentiginosis 96.60% DL
7 Moynahan syndrome 96.40% DL
8 rhabdoid tumor 96.20% DL
9 osteopathia striata-pigmentary dermopathy-white forelock syndrome 96.06% DL
10 congenital multiple café-au-lait macules-increased sister chromatid exchange syndrome 96.06% DL
11 acromelanosis 96.06% DL
12 X-linked lymphoproliferative disease due to SH2D1A deficiency 95.94% DL
13 leukonychia totalis-acanthosis-nigricans-like lesions-abnormal hair syndrome 95.86% DL
14 renal-hepatic-pancreatic dysplasia 95.75% DL
15 Joubert syndrome with renal defect 95.18% DL
16 peripheral nerve schwannoma 95.06% DL
17 karyomegalic interstitial nephritis 95.04% DL
18 schwannoma of twelfth cranial nerve 94.63% DL
19 thoracic malformation 94.62% DL
20 sympathetic neurilemmoma 94.61% 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.