Risdiplam

Evidence Level: L5 Predicted Indications: 50

Quick Overview

Item Value
Drug Name Risdiplam
DrugBank ID DB15305
Brand Names (EU) Evrysdi
Evidence Level L5
Predicted Indications 50
Top Prediction Score 99.45%

Approved Indication (EMA)

Evrysdi is indicated for the treatment of 5q spinal muscular atrophy (SMA) in patients with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies.  


Predicted New Indications

TxGNN model predictions for potential drug repurposing:

Rank Indication Score Source
1 acne (disease) 99.45% DL
2 drug-induced osteoporosis 97.72% DL
3 zinc, elevated plasma 97.32% DL
4 common wart 97.21% DL
5 metastatic melanoma 96.84% DL
6 dermatitis 96.62% DL
7 non-cutaneous melanoma 96.18% DL
8 acrodermatitis chronica atrophicans 96.11% DL
9 epithelioid cell melanoma 95.93% DL
10 eyelid melanoma 95.88% DL
11 scrotum melanoma 95.75% DL
12 neonatal dermatomyositis 95.74% DL
13 multiple endocrine neoplasia 95.58% DL
14 choroideremia 95.51% DL
15 acne keloid 95.40% DL
16 CDK4 linked melanoma 95.39% DL
17 superficial spreading melanoma 95.39% DL
18 malignant melanoma of the mucosa 95.39% DL
19 nodular malignant melanoma 95.39% DL
20 amelanotic skin melanoma 95.39% 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.