Avatrombopag
| Evidence Level: L5 | Predicted Indications: 10 |
Table of Contents
- Avatrombopag
- Avatrombopag: From a Thrombopoietin Receptor Agonist to Marcothrombocytopenia with Mitral Valve Insufficiency
Avatrombopag: From a Thrombopoietin Receptor Agonist to Marcothrombocytopenia with Mitral Valve Insufficiency
One-Sentence Summary
Avatrombopag is a thrombopoietin (TPO) receptor agonist that stimulates platelet production, and it is currently marketed in the United States. The TxGNN model predicts it may be effective for marcothrombocytopenia with mitral valve insufficiency, with a top score of 99.995%. There are currently 0 clinical trials and 0 publications supporting this prediction, so it is a research question, not an evidence-backed candidate.
Quick Overview
| Item | Content |
|---|---|
| Predicted New Indication | Marcothrombocytopenia with mitral valve insufficiency |
| TxGNN Prediction Score | 99.995% (model rank 254) |
| Evidence Level | L5 (model prediction only) |
| US Market Status | ✓ Marketed |
| Number of NDAs | 2 |
| Recommended Decision | Hold |
The evidence pack contains no approved-indication text for either NDA, so the original indication is not listed here.
Why is This Prediction Reasonable?
Avatrombopag is a TPO receptor agonist. It stimulates megakaryocyte proliferation and platelet production. Detailed DrugBank mechanism-of-action data are not available in the evidence pack, so this description comes from the mechanistic notes attached to the predictions.
Raising platelet count is a plausible strategy for a condition defined by low platelets. However, marcothrombocytopenia is likely genetic in origin, and the response to a TPO receptor agonist would depend on the specific defect. No supporting data were provided, so the mechanistic link is not verified.
The other nine predictions are weaker:
- Thrombocytopenia-related (ranks 2–3): “Hereditary thrombocytopenia with normal platelets” is plausible in principle, but the disease name is ambiguous and the mapping should be checked. “Transient neonatal thrombocytopenia” is self-limiting, and no neonatal safety data were provided, so the risk-benefit balance is unfavorable.
- Dense granule disease (rank 4): This is a platelet function defect, not a low platelet count. A TPO receptor agonist would not be expected to correct it, and the score looks like a knowledge-graph proximity artifact.
- Motor neuron and cortical disorders (ranks 5–10): These include ALS, its susceptibility entry, lower motor neuron syndrome, Mills syndrome, monomelic amyotrophy and polymicrogyria. No mechanistic link to TPO receptor agonism was found, and they likely reflect graph-structure artifacts. The ALS-related entries overlap and should not be counted as independent support.
Clinical Trial Evidence
Currently no related clinical trials registered.
Literature Evidence
Currently no related literature available.
US Market Information
| Authorization Number | Product Name | Dosage Form | Manufacturer |
|---|---|---|---|
| NDA210238 | DOPTELET | Tablet, film coated (oral) | AkaRx, Inc. |
| NDA219696 | Doptelet Sprinkle | Granule | AkaRx, Inc. |
Safety Considerations
Please refer to the package insert for safety information.
Conclusion and Next Steps
Decision: Hold
Rationale: The prediction has no clinical trial or literature support (L5), and the mechanistic link to this ultra-rare, likely genetic condition is unverified. Blocking safety gaps also remain, because package insert warnings and contraindications have not been retrieved.
To proceed, the following is needed:
- Package insert warnings and contraindications (a blocking gap)
- DrugBank mechanism-of-action data
- Confirmation of the disease definition and ontology mapping for “marcothrombocytopenia with mitral valve insufficiency”, and of the ambiguous “hereditary thrombocytopenia with normal platelets”
- A targeted literature and trial search (PubMed, ClinicalTrials.gov, ICTRP) for TPO receptor agonists in inherited thrombocytopenias
- Genetic-subtype analysis to decide whether a TPO receptor agonist could plausibly work
- Route compatibility assessment, which is still pending
- For the neonatal indication, neonatal safety data before any further consideration
This report is for research reference only and does not constitute medical advice. Repurposing candidates require clinical validation before any use.
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.