Drug intelligence / Profile preview

valiloxybate

Development stage
Phase 1
Lead developer
Avadel Pharmaceuticals
Modality
Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
Administration
Oral
01

Overview

Valiloxybate is an orally available small molecule developed by XWPharma as a neuroprotectant and therapy for sleep disorders such as narcolepsy and Parkinson's disease. It acts primarily as a prodrug for gamma-hydroxybutyric acid (GHB), which is released upon hydrolysis in the body. The active metabolite GHB exerts its effects by binding to the GHB receptor and acting as a weak agonist at the GABA B receptor. This mechanism modulates neurotransmission in the central nervous system and produces psychoactive effects relevant to sleep regulation and neuroprotection[1][6]. Valiloxybate has been investigated in clinical trials up to phase II for indications including narcolepsy and Parkinson's disease[1][2][7].

Other names
valiloxibic acid4-((L-Valyl)oxy)butanoic acidValiloxybate [USAN]VALILOXYBATE [INN]L-Valine 3-carboxypropyl esterL-Valine3-carboxypropyl esterL-Valine-3-carboxypropyl ester4-[(2S)-2-amino-3-methylbutanoyl]oxybutanoic acidMS-23099MS23099MS 23099HY-137429HY137429HY 137429CS-0138627CS0138627CS 0138627
02

Targets

GABA-B (Gamma-aminobutyric acid receptor type B)Gamma-hydroxybutyrate receptor

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