Harmony Biosciences announced the FDA has approved Wakix (pitolisant) for the treatment of excessive daytime sleepiness (EDS) in adult patients with narcolepsy. Wakix is the first and only treatment approved for patients with narcolepsy that is not scheduled as a controlled substance by the U.S. Drug Enforcement Administration (DEA).
The FDA approval of Wakix was based on two multicenter, randomized, double-blind, placebo-controlled studies (HARMONY 1 and HARMONY 1bis). These studies included a total of 261 adult patients, 75% to 80% of whom had a history of cataplexy. The patients were randomized to receive Wakix, placebo, or active control. Treatment duration was eight weeks, with a three-week dose titration phase followed by a 5-week stable dose phase. In both studies, Wakix demonstrated a statistically significant improvement in EDS as measured by the Epworth Sleepiness Scale (ESS) score. In the placebo-controlled trials conducted in patients with narcolepsy with or without cataplexy, the most common adverse reactions (occurring in ≥5% of patients and at twice the rate of placebo) with the use of Wakix were insomnia (6%), nausea (6%), and anxiety (5%).
Wakix was granted orphan drug designation for the treatment of narcolepsy in 2010.
About Wakix (pitolisant)
Wakix is a selective histamine 3 (H₃) receptor antagonist/inverse agonist. The mechanism of action of Wakix is unclear; however, its efficacy could be mediated through its activity at H₃ receptors, thereby increasing the synthesis and release of histamine, a wake promoting neurotransmitter.
Wakix was designed and developed by Bioprojet Societe Civile de Recherche (Bioprojet), who has marketed the product in Europe since its approval by the European Medicines Agency in 2016. Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States.
Narcolepsy is a rare, chronic, debilitating neurologic disorder of sleep-wake state instability that impacts up to 200,000 Americans and is primarily characterized by excessive daytime sleepiness (EDS) and cataplexy – its two cardinal symptoms – along with other manifestations of REM sleep dysregulation, which intrude into wakefulness. EDS is the inability to stay awake and alert during the day and is the symptom that is present in all people living with narcolepsy. In most patients, narcolepsy is caused by the loss of hypocretin, a neuropeptide in the brain that supports sleep-wake state stability. This disorder affects men and women equally, with typical symptom onset in adolescence or young adulthood; however, it can take up to a decade to be properly diagnosed.