#Eds and cataplexy full
Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment (see full prescribing information).
WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation.Patients meeting the ICSD-2 criteria for narcolepsy (with or without cataplexy) and an ESS score of fourteen or more enrolled in Harmony 1.
Harmony CTP registered patients aged 18 years and above who met the International Classification of Sleep Disorders (ICSD-2) criteria for narcolepsy with cataplexy with at least three cataplexy attacks in a week. Clinical trials on WAKIXįDA approval of WAKIX was based on the two multi-centre, randomised, double-blind, placebo-controlled trials, HARMONY CTP and HARMONY 1. WAKIX binds to H 3 receptors selectively and increases synthesis and release of histamine, a neurotransmitter that promotes wakefulness in the brain. The mechanism of action of WAKIX is unclear, however, its efficacy can be established through its activity on H 3 receptors. Pitolisant is a potent and highly selective antagonist or inverse agonist of the histamine-3 (H 3) receptor. All narcolepsy patients show excessive daytime sleepiness, while approximately two-thirds of them experience catalepsy. It ranges from moderate symptoms, such as drooping of eyelids, to severe, such as buckling of the knee or complete collapse of the body.Ĭataplexy may have a profound effect on the ability of a person to perform normal functions. It is an attribution of REM sleep dysregulation, causing wakefulness. The FDA approved WAKIX (pitolisant) for the treatment of EDS in adult patients with narcolepsy in August 2019 and extended the label for the treatment of cataplexy in adult narcolepsy patients in October 2020.Ĭataplexy is one of the physical manifestations of narcolepsy, the most debilitating chronic, rare neurological condition, characterised by abrupt, transient lack of muscle tone, often induced by strong feelings such as excitement or laughter. Harmony submitted an original new drug application (NDA) for pitolisant to the FDA for the treatment of EDS and cataplexy in adult patients with narcolepsy in December 2018 and the application was accepted for review in February 2019. The US Food and Drug Administration (FDA) provided orphan designation to pitolisant for narcolepsy in 2010, while breakthrough therapy designation to the drug for cataplexy in patients with narcolepsy was granted in April 2018. Harmony holds an exclusive authorisation from Bioprojet for the development, manufacturing and commercialisation of pitolisant in the US. The drug is marketed or distributed under a compassionate use programme in nine countries in the European Union (EU), including Germany, France, Netherlands, Italy, Belgium, Ireland, UK, Switzerland and Spain.