Thiamine

Evidence Level: L5

Predicted Indications: 3

Thiamine

Pharmacist Assessment Report

Thiamine: From an Unspecified Original Indication to resistance to thyroid hormone due to a mutation in thyroid hormone receptor beta

One-Sentence Summary

Thiamine (vitamin B1) is marketed in India mainly as injectable products, but the data compiled for this report contain no approved indication text, so its original licensed use cannot be stated. The TxGNN model ranks resistance to thyroid hormone due to a mutation in thyroid hormone receptor beta first (score 0.9943) and primary hereditary glaucoma second. This report focuses on open-angle glaucoma, the only candidate with any published literature, where 0 clinical trials and 6 publications were found. Most of these publications are observational or only tangentially related.

Quick Overview

Item	Content
Original Indication	Not available (no approved indication text in the licence records)
Predicted New Indication	Top-ranked: resistance to thyroid hormone due to a mutation in thyroid hormone receptor beta. Best-supported: open-angle glaucoma
TxGNN Prediction Score	0.9943 (top-ranked); 0.9936 (open-angle glaucoma)
Evidence Level	L5 (see note below)
India Market Status	Listed in a compiled Indian medicine price dataset (at least 20) – CDSCO approval status is not included in this data
Number of Listed Products	at least 20
Recommended Decision	Hold

Note on evidence level: The open-angle glaucoma candidate has one 1979 observational study that directly examines thiamine in chronic open-angle glaucoma, but it only compares blood levels. It does not test thiamine as a treatment. The other papers address glaucoma biology, niacin, or nutrient intake generally. No cited study evaluates thiamine as a treatment for the predicted disease, so the ceiling rule gives L5.

Why is This Prediction Reasonable?

Currently, detailed mechanism of action data is not available. Thiamine is a vitamin that acts as a cofactor for enzymes of carbohydrate metabolism, such as pyruvate dehydrogenase and transketolase. This role in cellular energy production is general background knowledge rather than data compiled for this report.

Open-angle glaucoma: The link is plausible but indirect. Mitochondrial dysfunction and oxidative stress are implicated in retinal ganglion cell loss and trabecular meshwork dysfunction. A 1979 study of 38 glaucoma patients and 12 controls reported significantly lower blood thiamine in the patients (P < 0.001). This shows an association with nutritional status only. It does not show that giving thiamine changes intraocular pressure or disease course.

Top-ranked and second-ranked predictions: No mechanistic link is identifiable for thyroid hormone resistance, which is a monogenic disorder of the receptor that thiamine is not known to affect. Primary hereditary glaucoma is driven mainly by genetic defects in aqueous outflow development, which thiamine is not known to correct. Neither candidate has any trial or publication, so these predictions rest on the TxGNN model (deep learning) score alone.

Clinical Trial Evidence

Currently no related clinical trials registered for any of the three predicted indications.

Literature Evidence

For open-angle glaucoma (abstracts were truncated, so the summaries below rely on titles and the visible text):

PMID	Year	Type	Journal	Key Findings
485004	1979	Observational	Annals of Ophthalmology	Patients with chronic open-angle glaucoma (n=38) had significantly lower blood thiamine than controls (n=12); no difference in ascorbic acid; poor thiamine absorption noted
32214001	2020	Cross-sectional	Nutrients	Korean national survey on obesity, nutrient supply and primary open-angle glaucoma
29565276	2018	Cross-sectional	Nutrients	Dietary niacin (not thiamine) and open-angle glaucoma in Korean survey data
40402521	2025	Metabolomic profiling	Investigative Ophthalmology & Visual Science	Aqueous humor metabolomics in glaucoma patients; metabolites with anti-inflammatory and neuroprotective potential tested in mice
30025119	2018	Molecular profiling	Investigative Ophthalmology & Visual Science	microRNA profiles in glaucomatous optic neuropathy
41889963	2026	Preclinical (preprint)	bioRxiv	MIF-CD74 signaling in trabecular meshwork dysfunction in glaucoma

India Market Information

At least 20 licences are recorded for single-ingredient thiamine products (the list may be partial). None of the records include approved indication text. Five examples:

Authorization Number	Product Name	Dosage Form	Approved Indication
136127	Macraberin Forte Injection	Vial of 2 ml injection	Not stated in records
218000	Thi 100mg Injection	Vial of 2 ml injection	Not stated in records
157754	Nexmin 100mg Injection	Vial of 2 ml injection	Not stated in records
28629	B Colex Injection	Vial of 1 ml injection	Not stated in records
248276	Zeba Syrup	Bottle of 200 ml syrup	Not stated in records

Nearly all listed products are injectables. The only exception is one oral syrup.

Safety Considerations

Please refer to the package insert for safety information.

Conclusion and Next Steps

Decision: Hold

Rationale: The TxGNN scores are very high (about 0.9940), but no clinical trial supports any of the three predictions. For open-angle glaucoma the literature shows only an association between low blood thiamine and disease, which is not evidence of treatment benefit. The two higher-ranked predictions have no identifiable mechanistic link.

To proceed, the following is needed:

Package insert warnings, contraindications and approved indications for the India licences
Mechanism of action data from DrugBank
A controlled study or well-designed trial of thiamine supplementation in open-angle glaucoma (intraocular pressure and visual field outcomes)
Review of full abstracts and full texts, since this assessment relied on truncated abstracts
Route and formulation assessment, since the marketed products are mostly injectables
Disclaimer

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