Elafibranor
| Evidence Level: L5 | Predicted Indications: 10 |
Table of Contents
Elafibranor: From Investigational (Unlicensed) Status to Amenorrhea (Disease)
One-Sentence Summary
Elafibranor is an investigational PPARα/δ dual agonist that has no EU marketing authorization and no confirmed original indication on record. The TxGNN model’s top-ranked prediction (of 10 candidates) suggests possible relevance to Amenorrhea (disease), with a 99.86% prediction score but zero supporting clinical trials and zero publications, placing this at the lowest evidence tier (L5).
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Not available — no EU marketing authorization or approved indication on record (investigational compound) |
| Predicted New Indication | Amenorrhea (disease) |
| TxGNN Prediction Score | 99.86% |
| Evidence Level | L5 |
| EU Market Status | Not Marketed |
| Number of Authorizations | 0 |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Currently, detailed mechanism-of-action data is not available from DrugBank in structured form. Based on the model’s own repurposing rationale, Elafibranor is understood to act as a PPARα/δ (peroxisome proliferator-activated receptor alpha/delta) dual agonist, working primarily through lipid metabolism and anti-inflammatory pathways.
However, the evidence pack’s mechanistic assessment explicitly states there is no known biological link between PPARα/δ signaling and the hypothalamic-pituitary-ovarian axis that governs amenorrhea. The high TxGNN score is attributed to knowledge-graph relational similarity rather than direct pharmacological or clinical support.
Because Elafibranor has no approved indication and no marketed formulation in this dataset, there is no established “original indication → new indication” pharmacological bridge to draw on here — unlike typical repurposing cases where mechanistic overlap between a known and predicted indication supports the hypothesis. All 10 predicted indications for this drug (including squamous cell carcinoma subtypes, Paget disease, and several rare congenital disorders) are rated L5 and flagged by the model itself as lacking direct biological rationale.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
Currently no related literature available.
EU Market Information
No EU marketing authorization found. Elafibranor is not currently marketed in the EU (0 licenses on record in this dataset).
Safety Considerations
Please refer to the SmPC for safety information. (No key warnings, contraindications, or drug-drug interaction data are available in the current evidence pack; a DDI database query returned no results.)
Conclusion and Next Steps
Decision: Hold
Rationale: The top prediction (amenorrhea) is explicitly flagged by the model’s own rationale as lacking mechanistic support, and is backed by no clinical trials or literature. Combined with the drug having no EU marketing authorization and no confirmed original indication, there is insufficient basis to advance this candidate.
To proceed, the following is needed:
- Confirmed mechanism-of-action data (DG002) via DrugBank/primary literature
- TFDA/EMA label warnings and contraindications (DG001) — currently blocking safety pre-screening
- Preclinical or mechanistic studies linking PPARα/δ agonism to reproductive endocrine function, if this indication is to be pursued further
- Clarification of Elafibranor’s regulatory/development status (e.g., ongoing trials in other indications such as NASH/PBC) to establish a credible original-indication baseline for comparison
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.