Elafibranor
| Evidence Level: L5 | Predicted Indications: 1 |
Table of Contents
Elafibranor: Amenorrhea (disease)
One-Sentence Summary
Elafibranor (Iqirvo) is a dual PPAR-alpha/delta agonist, developed for non-alcoholic steatohepatitis (NASH) and later repositioned as a treatment for primary biliary cholangitis (PBC), the indication on which it was approved by the FDA and EMA in 2024. The TxGNN model predicts it may be effective for amenorrhoea, with a very high score of 0.9986. Currently there are no clinical trials and no publications supporting this prediction, so it rests on model output alone.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Not recorded in the local data |
| Predicted New Indication | Amenorrhea (disease) |
| TxGNN Prediction Score | 0.9986 |
| 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?
Elafibranor is a dual agonist of PPAR-alpha and PPAR-delta (PPARA and PPARD). Its active metabolite, GFT-1007, is formed in the liver. Preclinical work showed reduced hepatic fat accumulation, inflammation and fibrosis. Detailed mechanism of action data are not otherwise available in the data compiled for this report, so the pharmacology above comes from target-ligand records only.
No direct mechanistic link between elafibranor and amenorrhoea has been established. PPAR signalling has been studied in ovarian and metabolic function, but any link to amenorrhoea is speculative. The very high TxGNN score is a computational prediction and is not supported by trials or literature. The relationship between the original indication (a cholestatic liver disease) and amenorrhoea has not been assessed.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
Currently no related literature available.
Australia Market Information
| ARTG Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| ARTG 15084X | Iqirvo | Tablet 80 mg (oral) | Not recorded in the data compiled for this report |
Safety Considerations
- Drug Interactions: No drug-drug interactions were identified. The two records returned only describe the drug’s pharmacological targets, PPAR-alpha and PPAR-beta/delta.
- Pharmacological note: PPAR-delta activation has context-dependent, sometimes pro-tumorigenic, effects reported in the literature. This warrants caution if oncology uses were ever explored.
Please refer to the TGA-approved Product Information (PI) for warnings and contraindications, as these were not available in the data compiled for this report.
Conclusion and Next Steps
Decision: Hold
Rationale: The prediction for amenorrhoea has a high model score but no clinical trials, no literature and no plausible established mechanism, so evidence is at the lowest level (L5). It is not appropriate to progress at this stage.
To proceed, the following is needed:
- Retrieval of the TGA/ARTG Product Information, including approved indication, warnings and contraindications
- Detailed mechanism of action data, and a literature review of PPAR-alpha/delta signalling in reproductive endocrine function
- Preclinical or observational evidence linking elafibranor to amenorrhoea (or the underlying causes)
- A review of the safety implications in women of reproductive age, including pregnancy and hormonal effects
- Assessment of the other ranked predictions, noting that the PPAR-delta link to tumour biology needs particular caution
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.