4-Aminohexanoic acid

4-Aminohexanoic acid is an investigative small molecule drug. Its primary mechanism of action involves the inhibition of 4-aminobutyrate aminotransferase (ABAT), a mitochondrial enzyme crucial for the catabolism of gamma-aminobutyric acid (GABA). By competitively binding to the active site of ABAT, 4-aminohexanoic acid prolongs GABAergic neurotransmission, which suggests its potential utility as an antiepileptic agent. Prodrug formulations of 4-aminohexanoic acid are currently undergoing Phase I trials, with the aim of enhancing cerebrospinal fluid concentrations for neurotherapeutic applications. Beyond its potential as a therapeutic, it also finds use in biochemical research, such as improving the proteolytic resistance of peptides and serving as a tracer in studies of GABA shunt activity.

Other names

g-aminocaproic acidHexanoic acid, 4-amino-, hydrochloride