Cysteamine
| Evidence Level: L5 | Predicted Indications: 10 |
Table of Contents
- Cysteamine
- Cysteamine: From Cystinosis to Mitochondrial Oxidative Phosphorylation Disorder Due to Nuclear DNA Anomalies
Cysteamine: From Cystinosis to Mitochondrial Oxidative Phosphorylation Disorder Due to Nuclear DNA Anomalies
One-Sentence Summary
Cysteamine is a cystine-depleting drug marketed in the US for cystinosis, sold as Cystagon, Procysbi and Cystaran. The TxGNN model predicts it may be effective for mitochondrial oxidative phosphorylation disorder due to nuclear DNA anomalies. There are currently 0 clinical trials and 0 publications supporting this specific prediction, so it rests on the model score alone.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Cystinosis (inferred from the marketed products, since the license records supplied no indication text) |
| Predicted New Indication | Mitochondrial oxidative phosphorylation disorder due to nuclear DNA anomalies |
| TxGNN Prediction Score | 99.93% |
| Evidence Level | L5 |
| US Market Status | ✓ Marketed |
| Number of NDAs | 9 |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Currently, detailed mechanism of action data is not available in the record. Cysteamine is known to lower cystine inside lysosomes. Its efficacy in cystinosis is established, and mechanistically it may be applicable to mitochondrial disease.
The only mechanistic link proposed is cysteamine’s antioxidant and glutathione-supporting activity, which could matter in mitochondrial dysfunction. This is plausible but unverified. The supplied record contains no trials, papers or preclinical data testing it. The high score probably reflects shared network neighbors in the knowledge graph rather than direct evidence.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
Currently no related literature available.
US Market Information
| Authorization Number | Product Name | Dosage Form | Manufacturer |
|---|---|---|---|
| NDA200740 | CYSTARAN | Solution | Leadiant Biosciences, Inc. |
| NDA213491 | PROCYSBI | Delayed-release granule | Horizon Therapeutics USA, Inc. |
| NDA020392 | Cystagon | Capsule | Mylan Pharmaceuticals Inc. |
Safety Considerations
Please refer to the package insert for safety information.
Conclusion and Next Steps
Decision: Hold
Rationale: This prediction is supported only by the model score, with no trials, literature or established mechanism for mitochondrial disease. It does not justify further investment yet.
To proceed, the following is needed:
- A targeted literature search and preclinical evidence for cysteamine (or glutathione-related mechanisms) in nuclear-DNA-related mitochondrial disorders
- Detailed mechanism of action data
- Package insert warnings and contraindications, which are currently missing and block safety screening
- Route and formulation compatibility assessment, which is still pending
Note on other predictions for this drug: The pack’s stronger signals lie elsewhere. Ocular cystinosis (rank 3) has L1 evidence, including Phase 3 topical cysteamine data. However, it is effectively an established use rather than a new repurposing finding. Lysosomal amino acid transport disorders (rank 2) have only indirect support. Both are better evaluated as separate reports.
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.