Drug intelligence / Profile preview

glyceryl triacetate

Development stage
Phase 2
Lead developer
Shaare Zedek Medical Center
Modality
Small Molecules
Administration
Oral, Topical
01

Overview

Glyceryl triacetate, also known as triacetin, is a triglyceride compound consisting of glycerol esterified with three acetic acid groups. In the clinical context of Canavan disease, it functions as a metabolic prodrug that provides a supplemental source of acetate to the brain. Canavan disease is a leukodystrophy caused by mutations in the ASPA gene, leading to a deficiency of the enzyme aspartoacylase. This deficiency results in the accumulation of N-acetylaspartate (NAA) and a critical shortage of acetate, which is essential for the synthesis of myelin lipids. By providing exogenous acetate, glyceryl triacetate aims to support myelination and mitigate the neurological decline associated with the disease. Beyond its investigational use in metabolic disorders, it is widely utilized as a food additive, plasticizer, and has historical applications as a topical antifungal agent.

Brand names
EnzacetinFungacetin
Other names
triacetinglycerin triacetate1,2,3-propanetriol triacetate1,2,3-triacetoxypropane

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