Ornithine
| Evidence Level: L5 | Predicted Indications: 10 |
Table of Contents
Ornithine: From Parenteral Nutrition Component to Congenital Prothrombin Deficiency
One-Sentence Summary
Ornithine is an amino acid in the urea cycle and polyamine pathway. In South Africa it is registered in two infusion products (Numeta G13E and G16E). The TxGNN model predicts it may be relevant to congenital prothrombin deficiency, but only 1 clinical trial is linked, and it is not a treatment study. There is no supporting literature, and the mechanistic link is judged implausible, so the high score is not backed by biological evidence.
Quick Overview
| Item | Content |
|---|---|
| Predicted New Indication | Congenital prothrombin deficiency |
| TxGNN Prediction Score | 97.52% |
| Evidence Level | L5 (model prediction only) |
| South Africa Market Status | Marketed |
| Number of SAHPRA Registrations | 2 |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Currently, detailed mechanism of action data is not available for ornithine. Ornithine is a urea cycle and polyamine pathway amino acid. It has no known role in the synthesis of coagulation factor II (prothrombin).
Congenital prothrombin deficiency is a rare inherited bleeding disorder caused by low or dysfunctional factor II. No plausible biological link to ornithine has been identified. The 97.52% score reflects patterns in the knowledge graph, not demonstrated biology, so it should be read as a hypothesis-generating signal only.
Clinical Trial Evidence
| Trial Number | Phase | Status | Enrollment | Key Findings |
|---|---|---|---|---|
| NCT04612764 | N/A | Recruiting | 62 | Cross-sectional study of liver fibrosis biomarkers (serum markers, Fibroscan, MRE) in urea cycle disorders. It does not test ornithine for prothrombin deficiency, so the overlap is only a general liver or metabolic theme. |
Literature Evidence
Currently no related literature available.
South Africa Market Information
| Registration Number | Product Name | Dosage Form |
|---|---|---|
| Reg. No. 52/25/0739 | Numeta G13E | Infusion |
| Reg. No. 52/25/0740 | Numeta G16E | Infusion |
Both registrations are for injectable (infusion) products. The registration records do not include approved indication text.
Safety Considerations
Please refer to the SAHPRA-approved Professional Information (PI) for safety information. Report adverse drug reactions to SAHPRA.
Conclusion and Next Steps
Decision: Hold
Rationale: The prediction rests on a model score alone. The only linked trial is an unrelated observational study, there is no literature, and no plausible mechanism connects ornithine to prothrombin synthesis. The other nine predicted indications in this pack (for example vitamin deficiency disorder, aortic malformation and acne) are also on Hold, with either no evidence or only indirect or unfavourable mechanistic evidence.
To proceed, the following is needed:
- A credible biological rationale linking ornithine to prothrombin (factor II) synthesis or function
- Preclinical or clinical data testing ornithine in congenital prothrombin deficiency
- Mechanism of action data from DrugBank
- SAHPRA Professional Information (PI) for the Numeta products, to complete the safety review
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.