Deferiprone
| Evidence Level: L5 | Predicted Indications: 8 |
Table of Contents
Deferiprone: oral iron chelator → hepatic porphyria
One-Sentence Summary
Deferiprone is an oral iron chelator supplied in Australia as a 1000 mg tablet (Ferriprox). The TxGNN model predicts it may be useful for hepatic porphyria, with a score of 0.9920. Support comes from 2 publications (one case-level preclinical report and one mouse study) and no registered clinical trials.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Not recorded in the local data |
| Predicted New Indication | hepatic porphyria |
| TxGNN Prediction Score | 0.9920 |
| Evidence Level | L5 |
| Australia Market Status | Listed on the PBS Schedule (1 of 1 items) – TGA (ARTG) registration status is not included in this data |
| Number of PBS Items | 1 (valid: 1) |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Detailed mechanism of action data are not available in the data compiled for this report. Deferiprone is known as an oral iron chelator, and the proposed rationale rests on that property.
Some porphyrias have iron-dependent pathology. In porphyria cutanea tarda, for example, excess hepatic iron promotes accumulation of uroporphyrin, and lowering labile iron could reduce this. This is the main mechanistic argument for the prediction.
The link is not general. Acute hepatic porphyrias are not iron-driven, so any benefit would probably be limited to specific subtypes. The supporting studies are also indirect: one is in congenital erythropoietic porphyria (not a hepatic form) and one is in a mouse model. Applicability to hepatic porphyria in humans is therefore not established.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 32678895 | 2020 | Preclinical / case-level | Blood | Iron chelation rescued haemolytic anaemia and skin photosensitivity in congenital erythropoietic porphyria |
| 17854053 | 2007 | Animal study (mouse) | Hepatology | In a mouse model of porphyria cutanea tarda, deferiprone was compared with iron-deficient diets for effects on hepatic uroporphyrin accumulation |
Australia Market Information
| ARTG Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| 11724C | Ferriprox | Tablet 1000 mg | Not recorded in the local data |
Safety Considerations
- Drug Interactions: 100 interactions were identified in the interaction screen. The 19 listed here are all rated Moderate, and the likely mechanism is reduced iron-chelator absorption or binding with polyvalent metal cations. They include:
- Calcium salts (phosphate, acetate, carbonate, citrate, glubionate, gluconate, lactate)
- Magnesium salts and antacids (oxide, carbonate, chloride, citrate, gluconate, hydroxide, sulfate, magaldrate)
- Aluminium hydroxide, attapulgite, kaolin and sucralfate
- Iron
For warnings and contraindications, please refer to the TGA-approved Product Information (PI).
Conclusion and Next Steps
Decision: Hold
Rationale: The prediction is scored highly by the model, but no cited study examines hepatic porphyria directly. The available literature covers a different porphyria (congenital erythropoietic) and a mouse model, so the evidence level is L5. The iron-driven mechanism may apply only to certain subtypes, such as porphyria cutanea tarda.
To proceed, the following is needed:
- Human studies in a specific iron-sensitive hepatic porphyria subtype, such as porphyria cutanea tarda
- Mechanism of action data for deferiprone
- Review of the TGA Product Information for warnings and contraindications
- Assessment of safety in porphyria populations, including interaction management with metal-containing products
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.