Introduction: Narcolepsy is a lifelong central nervous system (CNS) disorder characterized by excessive daytime sleepiness, cataplexy, disturbed nocturnal sleep, hypnagogic hallucinations, and sleep paralysis. Treatment is symptomatic and challenging. Current therapies with wake promoting agents, stimulants, and antidepressants improve symptoms but residual sleepiness or cataplexy may persist. Drug tolerance may develop. Adverse drug effects limit therapy. In the United States, sodium oxybate has been approved to treat daytime sleepiness and cataplexy in adults with narcolepsy since 2002. In 2018, it was approved for children ages 7-17 years with cataplexy with narcolepsy. Areas covered: This drug review includes an overview of narcolepsy, current pharmacotherapy, drug chemistry, pharmacodynamics, pharmacokinetics, and metabolism of sodium oxybate. Published results from 11 randomized control trials are reviewed. Databases searched included PubMed, Google Scholar, Lexi-Comp, Scopus, Science, and Ovid. Expert opinion: Sodium oxybate is an effective therapy for excessive daytime sleepiness and cataplexy in adults and children ages 7-17 years. It is also an effective therapy for disrupted nocturnal sleep. Sodium oxybate improves narcolepsy symptoms and enhances quality of life in narcolepsy patients.
Keywords: Cataplexy; daytime sleepiness; drug evaluation; narcolepsy; nocturnal sleep disruption; review; sodium 4-hydroxybutyrate; sodium oxybate.