Idebenone
| Evidence Level: L5 | Predicted Indications: 10 |
Table of Contents
Idebenone: From Leber Hereditary Optic Neuropathy to Hepatic Porphyria
One-Sentence Summary
Idebenone was originally used for Leber hereditary optic neuropathy. The TxGNN model predicts it may be effective for hepatic porphyria, but no clinical trials and no publications currently support this direction, so it is a computational prediction only.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Leber hereditary optic neuropathy (the Indian licence records contain no approved indication text) |
| Predicted New Indication | Hepatic porphyria |
| TxGNN Prediction Score | 0.9992 |
| Evidence Level | L5 |
| India Market Status | Listed in a compiled Indian medicine price dataset (7 of 7) – CDSCO approval status is not included in this data |
| Number of Listed Products | 7 (valid: 7) |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Currently, detailed mechanism of action data is not available. Based on known information, idebenone is a short-chain benzoquinone that acts as an electron carrier in the mitochondrial respiratory chain and as an antioxidant. Its use in Leber hereditary optic neuropathy, a mitochondrial disease, is the basis of its original indication.
A speculative link to hepatic porphyria is that antioxidant activity might reduce oxidative stress in hepatocytes. However, no data support a role for idebenone in heme biosynthesis defects, so this rationale cannot be verified.
The other top predictions are scored identically (0.9992 for several hepatic vascular and liver disorders). The score comes from the TxGNN model (deep learning) and may reflect similarities the model learned between related diseases, not biological evidence, rather than a disease-specific mechanism. Other predicted indications include hepatopulmonary syndrome, primitive portal vein thrombosis, hepatoportal sclerosis, idiopathic copper-associated cirrhosis, and several inflammatory myopathies. All of them also lack supporting evidence.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
Currently no related literature available.
India Market Information
| Authorization Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| 150824 | Norwayz Tablet | Strip of 10 tablets | Not stated |
| 108004 | IDEBEST 45MG TABLET | Strip of 10 tablets | Not stated |
| 49528 | Cebrol 45mg Tablet | Strip of 10 tablets | Not stated |
| 108716 | ID-45 Tablet | Strip of 10 tablets | Not stated |
| 28067 | Benovat 45mg Tablet | Strip of 10 tablets | Not stated |
Seven valid single-ingredient oral registrations exist in total; the five above are shown.
Safety Considerations
Please refer to the package insert for safety information.
Conclusion and Next Steps
Decision: Hold
Rationale: The prediction rests only on a model score, with no clinical trials or publications for hepatic porphyria. The proposed mechanism is speculative, and safety data are unavailable.
To proceed, the following is needed:
- Detailed mechanism of action data (MOA)
- Package insert warnings and contraindications for the Indian-marketed products
- Preclinical or mechanistic studies linking idebenone to heme biosynthesis or hepatic porphyria
- A literature and trial search specific to hepatic porphyria and the other top-ranked predictions
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.