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4-amino-3-hydroxybutyric acid (also known as GABOB) is a small molecule anticonvulsant and a derivative of the inhibitory neurotransmitter gamma-aminobutyric acid (GABA). Developed and manufactured by Haisen Pharmaceutical, it is approved for the treatment of epilepsy. 4-amino-3-hydroxybutyric acid acts as an agonist at GABA receptors, specifically showing affinity for GABA-B receptors, and may also inhibit 4-aminobutyrate aminotransferase (GABA transaminase), the enzyme responsible for GABA degradation. By enhancing GABAergic tone, it stabilizes neuronal excitability and prevents seizure activity. It is primarily administered orally and has been used clinically for several decades as an antiepileptic agent.
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