Imiglucerase

Evidence Level: L5 Predicted Indications: 50

Quick Overview

Item Value
Drug Name Imiglucerase
DrugBank ID DB00053
Brand Names (EU) Cerezyme
Evidence Level L5
Predicted Indications 50
Top Prediction Score 99.52%

Approved Indication (EMA)

Cerezyme (imiglucerase) is indicated for use as longterm enzyme replacement therapy in patients with a confirmed diagnosis of non-neuronopathic (Type 1) or chronic neuronopathic (Type 3) Gaucher disease who exhibit clinically significant nonneurological manifestations of the disease. The non-neurological manifestations of Gaucher disease include one or more of the following conditions: anaemia after exclusion of other causes, such as iron deficiency Thrombocytopenia Bone disease after exclusion


Predicted New Indications

TxGNN model predictions for potential drug repurposing:

Rank Indication Score Source
1 Hurler syndrome 99.52% DL
2 Gaucher disease 99.40% DL
3 Scheie syndrome 99.29% DL
4 benign neoplasm of adrenal gland 99.28% DL
5 autosomal ichthyosis syndrome with fatal disease course 99.24% DL
6 cholesteryl ester storage disease 99.12% DL
7 lysosomal storage disease with skeletal involvement 98.94% DL
8 Wolman disease with hypolipoproteinemia and acanthocytosis 98.93% DL
9 Wolman disease 98.78% DL
10 proximal myopathy with extrapyramidal signs 98.54% DL
11 growth hormone insensitivity syndrome with immune dysregulation 2, autosomal dominant 98.49% DL
12 Tay-Sachs disease 98.41% DL
13 familial apolipoprotein C-II deficiency 98.37% DL
14 adult Krabbe disease 98.36% DL
15 encephalopathy due to prosaposin deficiency 98.30% DL
16 Krabbe disease 98.15% DL
17 X-linked lymphoproliferative disease due to SH2D1A deficiency 98.13% DL
18 Cushing disease due to pituitary adenoma 98.09% DL
19 lysosomal acid lipase deficiency 97.98% DL
20 metachromatic leukodystrophy 97.97% 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.