Metreleptin

Evidence Level: L5 Predicted Indications: 53

Quick Overview

Item Value
Drug Name Metreleptin
DrugBank ID DB09046
Brand Names (EU) Myalepta
Evidence Level L5
Predicted Indications 53
Top Prediction Score 99.71%

Approved Indication (EMA)

Myalepta is indicated as an adjunct to diet as a replacement therapy to treat the complications of leptin deficiency in lipodystrophy (LD) patients: with confirmed congenital generalised LD (Berardinelli-Seip syndrome) or acquired generalised LD (Lawrence syndrome) in adults and children 2 years of age and above with confirmed familial partial LD or acquired partial LD (Barraquer-Simons syndrome), in adults and children 12 years of age and above for whom standard treatments have failed to achie

Predicted New Indications

TxGNN model predictions for potential drug repurposing:

Rank Indication Score Source
1 familial generalized lentiginosis 99.71% DL
2 gastrocutaneous syndrome 99.70% DL
3 Moynahan syndrome 99.67% DL
4 rhabdoid tumor 99.62% DL
5 osteopathia striata-pigmentary dermopathy-white forelock syndrome 99.62% DL
6 congenital multiple café-au-lait macules-increased sister chromatid exchange syndrome 99.61% DL
7 acromelanosis 99.61% DL
8 leukonychia totalis-acanthosis-nigricans-like lesions-abnormal hair syndrome 99.58% DL
9 benign neoplasm of adrenal gland 99.52% DL
10 peripheral nerve schwannoma 99.50% DL
11 schwannoma of twelfth cranial nerve 99.48% DL
12 sympathetic neurilemmoma 99.46% DL
13 trigeminal schwannoma 99.45% DL
14 microcystic/reticular schwannoma 99.45% DL
15 glaucoma 99.13% DL
16 lipoatrophic diabetes 99.06% DL
17 Gaucher disease 99.04% DL
18 proximal myopathy with extrapyramidal signs 99.04% DL
19 autosomal ichthyosis syndrome with fatal disease course 98.97% DL
20 pseudo-von Willebrand disease 98.96% DL

Showing top 20 of 53 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.