Deferiprone
| Evidence Level: L4 | Predicted Indications: 9 |
Table of Contents
Deferiprone: From Iron Chelation to Hepatic Porphyria
One-Sentence Summary
Deferiprone is an oral iron chelator marketed in the United States. The Evidence Pack does not list its approved indication text. The TxGNN model predicts it may be effective for hepatic porphyria, but the support is limited to 0 clinical trials and 2 preclinical publications (mouse models).
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Not provided in the Evidence Pack (indication fields are empty; deferiprone is generally recognized as an iron chelator for transfusional iron overload, to be verified against the label) |
| Predicted New Indication | Hepatic porphyria |
| TxGNN Prediction Score | 99.20% |
| Evidence Level | L4 |
| 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. Based on known information, deferiprone is an oral iron chelator, and its iron-lowering effect is established. Mechanistically, it may be applicable to iron-driven porphyrias.
In porphyria cutanea tarda, iron inhibits uroporphyrinogen decarboxylase, and reducing hepatic iron (for example by phlebotomy) is an effective treatment. Iron overload can also worsen other porphyrias. An oral chelator could therefore plausibly lower the iron burden and the accumulation of porphyrins.
The supporting data are preclinical. One mouse study of porphyria cutanea tarda compared deferiprone with iron-deficient diets. A second mouse study of congenital erythropoietic porphyria is only partially relevant, because that disease is erythropoietic rather than hepatic. No human clinical data were supplied.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 32678895 | 2020 | Preclinical (animal model) | Blood | Iron chelation rescued hemolytic anemia and skin photosensitivity in congenital erythropoietic porphyria (abstract truncated; based on title) |
| 17854053 | 2007 | Preclinical (murine model) | Hepatology | In a porphyria cutanea tarda mouse model (Hfe-/- mice given ALA), deferiprone was compared with iron-deficient diets for reducing hepatic uroporphyrin accumulation |
US Market Information
| Authorization Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| NDA021825 | FERRIPROX (Chiesi USA) | Film-coated tablet | Not listed in the data |
| NDA212269 | FERRIPROX (Chiesi USA) | Tablet | Not listed in the data |
| ANDA213239 | Deferiprone (Hikma) | Coated tablet | Not listed in the data |
| ANDA208800 | Deferiprone (Sun Pharmaceutical) | Tablet | Not listed in the data |
Safety Considerations
- Known Safety Issues: Agranulocytosis and neutropenia are the main deferiprone risks. They need to be weighed against the benefit in a rare disease.
Please refer to the package insert for other safety information.
Conclusion and Next Steps
Decision: Hold
Rationale: The evidence is at L4: the only support is two mouse studies, and one of them concerns an erythropoietic rather than hepatic porphyria. There are no clinical trials. The package insert warnings and contraindications are also missing, which blocks safety screening.
Among the other predictions, only beta-thalassemia (transfusional iron overload) and pyruvate kinase deficiency (secondary iron overload) have a plausible iron-chelation rationale. Beta-thalassemia is probably an on-label use rather than true repurposing. The remaining candidates have prediction scores only and no supporting mechanism.
To proceed, the following is needed:
- Package insert warnings, contraindications and approved indication text, to unblock safety screening
- Mechanism of action data (MOA), for example from DrugBank
- A targeted literature search for human data in porphyria cutanea tarda and other iron-sensitive porphyrias
- A risk-benefit plan for agranulocytosis and neutropenia monitoring in a rare-disease population
This report is for research reference only and does not constitute medical advice. Repurposing candidates require clinical validation before use.
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.