Ambrisentan

Evidence Level: L5

Predicted Indications: 5

Ambrisentan

Pharmacist Assessment Report

Ambrisentan: endothelin type A receptor antagonist → Pulmonary arteriovenous malformation

One-Sentence Summary

Ambrisentan is an endothelin type A receptor antagonist. The TxGNN model predicts it may be relevant to pulmonary arteriovenous malformation, but no clinical trials are registered and the only linked publication is 1 case report, which concerns pulmonary arterial hypertension rather than the malformation itself. This is a computational prediction only.

Quick Overview

Item	Content
Original Indication	Not recorded in the local data
Predicted New Indication	Pulmonary arteriovenous malformation
TxGNN Prediction Score	0.9941
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?

Ambrisentan is an endothelin type A receptor antagonist. Detailed mechanism of action data is not available in the data compiled for this report, so this description reflects the drug’s general class.

Pulmonary arteriovenous malformations are often associated with hereditary haemorrhagic telangiectasia (HHT) and involve dysregulated angiogenesis and vascular remodelling. Endothelin signalling is plausibly relevant to the associated pulmonary vascular disease, which is the basis for a possible mechanistic link.

However, this support is indirect. The only linked publication describes pulmonary arterial hypertension in a patient with HHT. It does not study treatment of the malformation itself. The high TxGNN score is a computational output and is not corroborated by direct studies.

Clinical Trial Evidence

Currently no related clinical trials registered.

Literature Evidence

PMID	Year	Type	Journal	Key Findings
33969094	2021	Case report	World Journal of Clinical Cases	Report of pulmonary arterial hypertension in a patient with HHT, with family gene analysis, to raise awareness of this co-occurrence. It does not evaluate ambrisentan for the malformation.

Australia Market Information

ARTG Number	Product Name	Dosage Form	Approved Indication
ARTG 12180C	Cipla Ambrisentan	Tablet 10 mg	Not recorded in the local data

Safety Considerations

Drug Interactions: Interaction screening returned 100 records. Those at moderate level include canagliflozin, dapagliflozin, empagliflozin, ertugliflozin, eliglustat and naltrexone. Those at minor level include clarithromycin, miconazole and omeprazole.
Systemic endothelin receptor blockade carries embryo-fetal toxicity concerns, so use in developmental or cosmetic conditions is hard to justify.

Please refer to the TGA-approved Product Information (PI) for full warnings and contraindications, as label-based data were not available for this report.

Conclusion and Next Steps

Decision: Hold

Rationale: No clinical trial or publication directly studies ambrisentan in pulmonary arteriovenous malformation. The prediction rests on a model score and an indirect mechanistic link, so it remains a research question.

To proceed, the following is needed:

Studies directly evaluating ambrisentan or other endothelin antagonists in pulmonary arteriovenous malformation, with or without HHT
Detailed mechanism of action data
Warnings and contraindications from the TGA Product Information, followed by a safety review for this population
Disclaimer

This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.