FDA Approves Wakix (pitolisant), a First-in-Class Medication for the Treatment of Excessive Daytime Sleepiness in Adult Patients with Narcolepsy

Harmony Biosciences, LLC (Harmony) announced today that the U.S. Food and Drug Administration (FDA) 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).

"We are extremely proud to bring Wakix to market for those living with narcolepsy, a chronic, debilitating, rare neurologic disorder," said Harmony’s Chairman and Chief Executive Officer, John C. Jacobs. "At Harmony we share a vision to develop novel treatment options for people living with rare diseases, with a focus on those that affect the central nervous system. The approval of Wakix strengthens our commitment to making that vision a reality."

Wakix, a first-in-class medication, is a selective histamine 3 (H₃) receptor antagonist/inverse agonist that works through a novel mechanism of action to increase the synthesis and release of histamine, a wake-promoting neurotransmitter in the brain. Wakix is administered orally once daily in the morning upon wakening.

"The approval of Wakix provides healthcare professionals managing people living with narcolepsy a new and important treatment option for their patients," said Harmony’s Chief Medical Officer, Jeffrey Dayno, M.D. "Additionally, Wakix is the only non-scheduled treatment option approved for adult patients with narcolepsy, and it offers an important benefit/risk profile to address the unmet medical need that exists in people living with narcolepsy."

The efficacy of Wakix for the treatment of EDS in adult patients with narcolepsy was evaluated in two multicenter, randomized, double-blind, placebo-controlled studies (HARMONY 1 and HARMONY 1bis). These studies included a total of 261 patients who were randomized to receive Wakix, placebo, or active control; these patients had a median age of 37 (HARMONY 1) and 40 (HARMONY 1bis). Treatment duration was eight weeks, with a three-week dose titration phase followed by a 5-week stable dose phase; 75% to 80% of the patients in these studies had a history of cataplexy. In both of these 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 will be commercially available to healthcare professionals and appropriate patients in the U.S. in the fourth quarter of 2019.

About Wakix (pitolisant)

Wakix is a first-in-class medication approved for use in the U.S. for the treatment of excessive daytime sleepiness in adult patients with narcolepsy. It was granted orphan drug designation for the treatment of narcolepsy in 2010. 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.

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