Ripasudil
| Evidence Level: L5 | Predicted Indications: 10 |
Table of Contents
- Ripasudil
- Ripasudil: From ROCK-Inhibitor Glaucoma Therapy (Unconfirmed in Taiwan) to Hyperprolactinemia
Ripasudil: From ROCK-Inhibitor Glaucoma Therapy (Unconfirmed in Taiwan) to Hyperprolactinemia
One-Sentence Summary
Ripasudil (DB13165) is a Rho-kinase (ROCK) inhibitor, pharmacologically known for lowering intraocular pressure by relaxing the trabecular meshwork — though no confirmed original indication or MOA data is on file for this dataset, and the drug is not currently marketed in Taiwan. The TxGNN model’s top-ranked prediction is Hyperprolactinemia, but this prediction is supported by no clinical trials and no literature, and the mechanistic rationale itself is flagged as weak. Several other candidates in the same prediction batch (glaucoma-spectrum diseases) show far stronger mechanistic plausibility, but likewise lack any supporting studies.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Not established in local regulatory filings (drug not marketed in Taiwan); known globally as a ROCK inhibitor used for glaucoma/ocular hypertension (per literature references cited in this evidence pack, unconfirmed here) |
| Predicted New Indication | Hyperprolactinemia (disease) |
| TxGNN Prediction Score | 99.87% |
| Evidence Level | L5 (model prediction only, no supporting trials or literature) |
| Taiwan Market Status | Not marketed (Not marketed) |
| Number of Registrations | 0 |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Detailed mechanism-of-action data for Ripasudil is not available in this evidence pack (flagged as a High-severity data gap). Based on the mechanistic notes embedded in the prediction rationale, Ripasudil is understood to be a Rho-kinase (ROCK) inhibitor, a drug class that acts primarily on smooth muscle relaxation and cytoskeletal regulation — most notably relaxing the trabecular meshwork to increase aqueous humor outflow, the basis for its known use in glaucoma/ocular hypertension in other markets.
For the top-ranked prediction, Hyperprolactinemia, the model’s own rationale acknowledges the mechanistic link is weak: prolactin secretion is regulated primarily via the dopamine D2 receptor pathway, and there is no established evidence that Rho/ROCK signaling plays a key role in pituitary lactotroph function. The high TxGNN score likely reflects indirect or noisy connections in the knowledge graph rather than a genuine pharmacological relationship.
Notably, this candidate batch contains several other predictions with substantially stronger mechanistic coherence — see the table below. Diseases involving impaired aqueous humor outflow (Axenfeld anomaly, hydrophthalmos, primary infantile glaucoma) align directly with Ripasudil’s known ROCK-inhibition/IOP-lowering mechanism, even though none currently have supporting clinical or literature evidence.
Other Candidate Indications (Same Prediction Batch)
| Disease | TxGNN Score | Evidence Level | Recommendation | Mechanistic Note |
|---|---|---|---|---|
| Hyperprolactinemia (disease) | 99.87% | L5 | Hold | Weak — no known ROCK/prolactin pathway link |
| Axenfeld anomaly | 99.86% | L5 | Research Question | Plausible — anterior chamber angle disorder overlaps with aqueous outflow mechanism |
| Hydrophthalmos | 99.85% | L5 | Research Question | Plausible — congenital glaucoma directly matches IOP-lowering mechanism |
| Glaucoma 3, primary infantile, B | 99.81% | L5 | Research Question | Plausible — shares core outflow-resistance pathology with adult glaucoma |
| Familial hyperprolactinemia | 99.79% | L5 | Hold | Weak, same rationale as rank 1 |
| Dense granule disease | 99.79% | L5 | Hold | Directionally uncertain — ROCK inhibition may impair rather than restore platelet function |
| Macrothrombocytopenia with mitral valve insufficiency | 99.77% | L5 | Hold | Directionally uncertain, no supporting data |
| Hereditary thrombocytopenia with normal platelets | 99.77% | L5 | Hold | Directionally uncertain, no supporting data |
| Transient neonatal thrombocytopenia | 99.77% | Pending | Pending | Not yet scored |
| Thrombocytopenia | 99.73% | L5 | Hold | One indirect paper (dengue-induced permeability, not Ripasudil-specific) |
Clinical Trial Evidence
(For top-ranked prediction: Hyperprolactinemia)
Currently no related clinical trials registered
Literature Evidence
(For top-ranked prediction: Hyperprolactinemia)
Currently no related literature available
For reference, the only literature found anywhere in this batch relates to rank 10 (Thrombocytopenia):
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 40189910 | 2025 | Mechanistic (in vitro) | Virulence | Shows Ripasudil and other ROCK/Src inhibitors reduce dengue virus (DENV2)-induced endothelial permeability in vitro; not a direct study of Ripasudil for thrombocytopenia and not generalizable as a therapeutic indication |
Taiwan Market Information
This drug is not currently marketed in Taiwan (Not marketed). No registration or license records are available in this dataset.
Safety Considerations
Please refer to the package insert for safety information.
(Note: TFDA label warnings/contraindications are flagged as a Blocking data gap in this evidence pack — they must be obtained before any S1 safety screening can proceed.)
Conclusion and Next Steps
Decision: Hold
Rationale: The top-ranked prediction (Hyperprolactinemia) has no supporting clinical trials or literature, and the mechanistic link itself is assessed as weak by the model’s own rationale. Combined with two Blocking/High-severity data gaps (missing TFDA safety label, missing MOA), there is insufficient basis to advance this candidate past S0.
To proceed, the following is needed:
- TFDA label data (warnings, contraindications) — currently Blocking (DG001)
- Confirmed mechanism of action from DrugBank — currently High severity (DG002)
- If pursuing repurposing further, consider redirecting research focus toward the glaucoma-spectrum candidates in this batch (Axenfeld anomaly, hydrophthalmos, primary infantile glaucoma), which have materially stronger mechanistic rationale despite currently lacking any clinical or literature support
- Original indication and regulatory status confirmation, since this drug is not currently marketed in Taiwan
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.