Pitolisant for the treatment of cataplexy in adults with narcolepsy

ABSTRACT Introduction Narcolepsy is a chronic neurological disorder of sleep-wake instability that typically requires lifelong pharmacotherapy. Narcolepsy medications target the cardinal symptoms of excessive daytime sleepiness (EDS), cataplexy (the sudden loss of muscle tone often triggered by strong emotion), or both. Areas covered A comprehensive review of the clinical trial data evaluating the efficacy of pitolisant (Wakix) for the treatment of cataplexy is provided. Expert opinion There remains an important need to improve the recognition and assessment of cataplexy. Treatment effectiveness should be evaluated with a patient-specific approach that takes into account not only a reduction in the frequency and severity of cataplexy attacks, but also fewer limitations imposed by cataplexy and enhancements in quality of life. Pitolisant, a histamine H3-receptor antagonist/inverse agonist, has demonstrated efficacy for the reduction of both cataplexy and EDS in patients with narcolepsy, including patients who experience frequent cataplexy attacks. Histamine plays an important role in the regulation of sleep and wakefulness, and pitolisant is thought to act directly via the histamine system and indirectly via other neurotransmitter systems. Based on the favorable efficacy and safety profile demonstrated in clinical trials, pitolisant should be considered as first-line treatment for patients with narcolepsy.