Elafibranor
| Evidence Level: L5 | Predicted Indications: 1 |
Table of Contents
Elafibranor: From Primary Biliary Cholangitis to Amenorrhea
One-Sentence Summary
Elafibranor (marketed in Canada as IQIRVO) is a dual PPARα/δ agonist. It is approved for primary biliary cholangitis (PBC) in other jurisdictions and was originally developed for nonalcoholic steatohepatitis (NASH). The TxGNN model predicts it may be effective for amenorrhea. There are currently 0 clinical trials and 0 publications supporting this direction, so the prediction rests on the model score alone.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Primary biliary cholangitis (based on the approvals described in pharmacology data; the Canadian licence record has no indication text) |
| Predicted New Indication | Amenorrhea |
| TxGNN Prediction Score | 0.9986 |
| Evidence Level | L5 |
| Canada Market Status | Marketed (marketed DINs: 1 of 1) |
| Number of DINs | 1 (marketed: 1) |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Detailed mechanism-of-action data is not available in the compiled data. Pharmacology data identifies elafibranor as a dual agonist of PPARα and PPARδ (PPARβ/δ). In the liver it is converted to its main active metabolite, GFT-1007. Preclinical work showed reduced hepatic fat accumulation, inflammation and fibrosis. The NASH programme was stopped in mid-2020 after an interim futility analysis. The drug was then repositioned for PBC, approved by the FDA in June 2024 and authorised by the EMA in September 2024.
The link between this profile and amenorrhea is speculative. PPAR signalling has theoretical connections to ovarian steroidogenesis, insulin sensitivity and metabolic regulation, which might matter in metabolic forms of amenorrhea such as PCOS-related amenorrhea. No trial, publication or mechanistic data in the compiled material supports this pathway. A high TxGNN model (deep learning) score is a hypothesis-generating signal, not clinical evidence.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
Currently no related literature available.
Canada Market Information
| DIN | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| 02557010 | IQIRVO | Not specified | Not specified |
Safety Considerations
- Drug Interactions: The interaction query completed, but it returned only pharmacology target records, not clinical drug–drug interactions. These records show that elafibranor acts on PPARα (PPARA) and PPARβ/δ (PPARD).
Please refer to the package insert for warnings and contraindications, as this information was not available in the compiled data.
Conclusion and Next Steps
Decision: Hold
Rationale: The only support for amenorrhea is a high model prediction score (0.9986). There are no clinical trials or publications, the mechanistic link is speculative, and the data on label warnings and contraindications is missing. This is Evidence Level L5.
To proceed, the following is needed:
- Health Canada product monograph warnings and contraindications, which are required before any safety screening
- Detailed mechanism-of-action data (for example from DrugBank)
- A literature and trial search on PPARα/δ agonism and amenorrhea, or on the related conditions (such as PCOS)
- Preclinical or mechanistic evidence for an effect on the reproductive endocrine axis
- Assessment of the clinical need in amenorrhea, the target population, and route and formulation compatibility
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.