Edaravone
| Evidence Level: L5 | Predicted Indications: 2 |
Table of Contents
- Edaravone
- Pharmacist Assessment Report
- Edaravone: From Ischaemic Stroke and ALS to Heparin Cofactor 2 Deficiency
- One-Sentence Summary
- Quick Overview
- Why is This Prediction Reasonable?
- Clinical Trial Evidence
- Literature Evidence
- Australia Market Information
- Safety Considerations
- Conclusion and Next Steps
- Disclaimer
Edaravone: From Ischaemic Stroke and ALS to Heparin Cofactor 2 Deficiency
One-Sentence Summary
Edaravone is a free radical scavenger originally used to treat ischaemic stroke and amyotrophic lateral sclerosis (ALS). The TxGNN model (deep learning) predicts it may be effective for heparin cofactor 2 deficiency, but this is a model prediction only, with 0 clinical trials and 0 publications supporting this specific indication.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Ischaemic stroke and amyotrophic lateral sclerosis (the Australian licence record contains no approved indication text) |
| Predicted New Indication | Heparin cofactor 2 deficiency |
| TxGNN Prediction Score | 0.9947 |
| 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 is not available in the data compiled for this report. Based on published knowledge, edaravone is a free radical scavenger that quenches hydroxyl radicals and inhibits lipid peroxidation. Its benefit in ischaemic stroke and ALS is thought to come from limiting oxidative injury to neurons.
Heparin cofactor 2 deficiency is a thrombophilia caused by reduced inhibition of thrombin. Edaravone does not act on the coagulation cascade, so no direct mechanistic link has been established. The high score comes from the TxGNN model (deep learning) and may reflect similarities the model learned between related diseases, not biological evidence. It should be read as a hypothesis-generating signal, not as evidence of benefit.
Other predictions in the top 10
| Rank | Predicted Indication | Score | Evidence Level | Comment |
|---|---|---|---|---|
| 2 | Factor 5 excess with spontaneous thrombosis | 0.9906 | L5 | Hypercoagulable state; no plausible link |
The motor neuron disease predictions (ranks 6, 7, 9 and 10) are the most biologically plausible. They sit within or next to the ALS spectrum for which edaravone is already approved, so they are extensions of an existing use rather than true repurposing.
Clinical Trial Evidence
Currently no related clinical trials registered for heparin cofactor 2 deficiency. No trials were identified for any of the top 10 predicted indications.
Literature Evidence
Currently no related literature available for heparin cofactor 2 deficiency.
For context, the literature retrieved for the ALS-related prediction (rank 6) is shown below. It is mechanistic or preclinical, and none of it is a clinical trial.
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 38474192 | 2024 | Review | Int J Mol Sci | Update on edaravone as an antioxidant. Developed in Japan for acute cerebral infarction, later applied to ALS, with expanding use in neurodegenerative diseases |
| 32241621 | 2020 | Review | Bioorg Med Chem | Chemical reactivity and uses of edaravone (PMP), also a laboratory reagent; notes approval for stroke and ALS |
| 36595221 | 2023 | Preclinical (in vitro) | FEBS Open Bio | Mouse stem cell-derived motor neurons are susceptible to ferroptosis, a lipid peroxidation-driven cell death |
| 41331940 | 2025 | Preclinical (zebrafish) | Acta Neuropathol Commun | Human TDP-43 overexpression in zebrafish motor neurons produces MND-like phenotypes |
| 40672281 | 2025 | Preclinical (zebrafish, preprint) | bioRxiv | Preprint version of the TDP-43 zebrafish study |
Australia Market Information
| ARTG Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| ARTG 14804E | Radicava | Solution concentrate for I.V. infusion, 30 mg in 20 mL | Not stated in the data compiled for this report |
Safety Considerations
No warnings, contraindications or drug interaction data were available in the data compiled for this report. Please refer to the TGA-approved Product Information (PI) for safety information.
Conclusion and Next Steps
Decision: Hold
Rationale: The heparin cofactor 2 deficiency prediction rests on the TxGNN model (deep learning) score alone, which may reflect similarities the model learned between related diseases, not biological evidence. There are no trials or publications, and there is no plausible mechanistic link between a free radical scavenger and a thrombin-inhibitor deficiency.
To proceed, the following is needed:
- Product Information warnings and contraindications for the Australian product, which are currently missing and block safety screening
- Mechanism of action data (for example from DrugBank)
- Approved indication text for the Australian licence
- Any preclinical or clinical evidence linking edaravone to the predicted thrombophilia
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.