Latanoprost

證據等級: L5

預測適應症: 10 個

Latanoprost: From Open-Angle Glaucoma to Primary Hereditary Glaucoma

One-Sentence Summary

Latanoprost is a prostaglandin F2α (FP) receptor agonist widely used to reduce intraocular pressure (IOP) in open-angle glaucoma and ocular hypertension. The TxGNN model predicts it may be effective for Primary Hereditary Glaucoma, with 1 completed Phase 2 clinical trial currently providing direct supporting evidence for this direction.

Quick Overview

Item	Content
Original Indication	Open-angle glaucoma / Ocular hypertension
Predicted New Indication	Primary Hereditary Glaucoma
TxGNN Prediction Score	99.88%
Evidence Level	L2
Italy Market Status	Not marketed
Number of Authorizations	0
Recommended Decision	Proceed with Guardrails

Why is This Prediction Reasonable?

Currently, detailed mechanism of action data is not available in the Evidence Pack. Based on known pharmacology, Latanoprost is a selective prostaglandin F2α (FP) receptor agonist that lowers intraocular pressure by increasing aqueous humor outflow through the uveoscleral pathway. Its efficacy and safety in reducing IOP for open-angle glaucoma and ocular hypertension are well established across decades of clinical use.

Primary hereditary glaucoma — caused by mutations in genes such as MYOC (myocilin) and CYP1B1 — shares the same pathological endpoint as open-angle glaucoma: elevated IOP leading to progressive optic nerve damage and irreversible vision loss. Although the root cause is genetic rather than acquired, IOP control remains the cornerstone of management for most hereditary glaucoma forms, including cases refractory to surgical procedures.

Because Latanoprost's mechanism acts downstream of the genetic defect — directly enhancing uveoscleral outflow regardless of the upstream mutation — its IOP-lowering effect is biologically applicable to this population. Notably, a Phase 2 trial (NCT01527682) directly evaluated a prostaglandin analogue and dorzolamide combination in paediatric hereditary glaucoma patients who had already failed surgery, providing the strongest currently available direct clinical evidence for this repurposing pathway.

Clinical Trial Evidence

Trial Number	Phase	Status	Enrollment	Key Findings
NCT01527682	Phase 2	Completed	37	Assessed ocular hypotensive efficacy and safety of a prostaglandin analogue (latanoprost class) plus dorzolamide in paediatric glaucoma patients refractory to surgery. Targeted 68 eyes across 34–68 patients; protocol was amended mid-study. Full-text review needed to confirm primary endpoint outcomes.

Literature Evidence

Currently no related literature available.

Italy Market Information

Latanoprost is not currently approved or marketed in Italy (AIFA). No authorization records are available.

Safety Considerations

Please refer to the package insert for safety information.

Conclusion and Next Steps

Decision: Proceed with Guardrails

Rationale: A completed Phase 2 trial directly evaluates prostaglandin analogue therapy in paediatric primary hereditary glaucoma refractory to surgery, and Latanoprost's FP receptor-mediated IOP reduction is mechanistically valid regardless of the underlying genetic aetiology — making this a well-grounded repurposing candidate.

To proceed, the following is needed:

Full-text review of NCT01527682 to confirm primary endpoint achievement, safety profile, and subgroup-specific outcomes
Mechanism of action (MOA) data from DrugBank API to complete the mechanistic rationale
Safety and contraindication data from the Italian / AIFA package insert to enable S1 safety screening
Paediatric dosing and formulation suitability assessment for the primary hereditary glaucoma population
Drug–drug interaction (DDI) profile review, particularly for patients on concurrent anti-glaucoma therapies
Disclaimer

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