Dorzolamide
| Evidence Level: L2 | Predicted Indications: 10 |
Table of Contents
Dorzolamide: From Glaucoma (Topical IOP Lowering) to Primary Hereditary Glaucoma
One-Sentence Summary
Dorzolamide is a topical carbonic anhydrase inhibitor eye drop that lowers eye pressure, and it is marketed in the US in generic ophthalmic solutions. The TxGNN model predicts it may be effective for primary hereditary glaucoma, but only 1 clinical trial (a Phase 2 study in pediatric glaucoma) and no publications currently support this specific indication.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Not stated in the US license records; the drug is used as topical therapy for glaucoma and ocular hypertension |
| Predicted New Indication | Primary hereditary glaucoma |
| TxGNN Prediction Score | 99.99% |
| Evidence Level | L2 |
| US Market Status | ✓ Marketed |
| Number of NDAs | 15 |
| Recommended Decision | Hold |
Why is This Prediction Reasonable?
Detailed mechanism of action data is not available in the input record. Dorzolamide is known to inhibit carbonic anhydrase II in the ciliary epithelium of the eye. This reduces aqueous humor secretion and lowers intraocular pressure (IOP).
Glaucoma is driven largely by elevated IOP, so an IOP-lowering drug fits any glaucoma subtype in principle, including hereditary and congenital forms. Because the mechanism acts on fluid production and not on the cause of the disease, it does not depend on why the pressure is high.
There is no direct evidence in hereditary or congenital forms beyond one trial, whose population is not fully confirmed. This is a plausible extension, not a proven one. The prediction appears to be a specific subtype of the drug’s existing glaucoma use, not a new therapeutic area.
Clinical Trial Evidence
| Trial Number | Phase | Status | Enrollment | Key Findings |
|---|---|---|---|---|
| NCT01527682 | Phase 2 | Completed | 37 | Latanoprost plus dorzolamide for pediatric glaucoma refractory to surgery. Primary aim is the ocular hypotensive effect, with safety also assessed. Study ran 2009-07 to 2016-11. No results are given in the record. |
The trial’s relevance is graded B. The title is truncated, so the exact population (hereditary versus other pediatric glaucoma) needs manual verification.
Literature Evidence
Currently no related literature available.
US Market Information
The US record lists 15 authorizations in total. All five listed here are generic ANDAs. The record gives no approved-indication text for any of them.
| Authorization Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| ANDA215660 | Dorzolamide Hydrochloride (Fosun Pharma USA) | Solution | Not stated in record |
| ANDA204778 | Dorzolamide Hydrochloride Ophthalmic Solution (Micro Labs) | Solution/drops | Not stated in record |
| ANDA078395 | Dorzolamide Hydrochloride (Advagen Pharma) | Solution | Not stated in record |
| ANDA202053 | Dorzolamide Hydrochloride Ophthalmic (Florida Pharmaceutical Products) | Solution | Not stated in record |
| ANDA078981 | Dorzolamide Hydrochloride (Sandoz) | Solution/drops | Not stated in record |
Safety Considerations
Please refer to the package insert for safety information.
Conclusion and Next Steps
Decision: Hold
Rationale: Support for hereditary glaucoma rests on a single completed Phase 2 trial with an unconfirmed population and no reported results. The package-insert safety review is also incomplete, which blocks moving to safety screening.
To proceed, the following is needed:
- Manual review of NCT01527682 (population, whether the carbonic anhydrase inhibitor is dorzolamide, and results)
- Package insert warnings and contraindications for the US products, especially for pediatric use
- Detailed mechanism of action data, plus a correction of the empty original-indication field in the record
- Note that other high-scoring predictions, such as open-angle glaucoma, already have Phase 3/4 and systematic-review evidence. These are the drug’s existing use, so they should not be reported as repurposing findings. Those entries also overlap and should be merged.
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.