FDA Okays Pitolisant (Wakix) for Narcolepsy

Megan Brooks


August 15, 2019

The US Food and Drug Administration (FDA) has approved pitolisant (Wakix, Harmony Biosciences) for the treatment of excessive daytime sleepiness (EDS) in adults with narcolepsy.

Pitolisant, 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. It is administered orally once daily in the morning upon waking.

"The approval of Wakix provides healthcare professionals managing people living with narcolepsy a new and important treatment option for their patients," Harmony's Chief Medical Officer Jeffrey Dayno, MD, said in a release.

"Additionally, Wakix is the only nonscheduled 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," said Dayno.

The efficacy of pitolisant was evaluated in two multicenter, randomized, double-blind, placebo-controlled studies involving 261 patients with narcolepsy with or without cataplexy. Treatment lasted 8 weeks, with a 3-week dose titration phase followed by a 5-week stable-dose phase.

In both studies, pitolisant demonstrated a statistically significant improvement in EDS, as measured by the Epworth Sleepiness Scale score, the company said.

The most common adverse reactions (occurring in ≥ 5% of patients and at twice the rate of placebo) with the use of pitolisant were insomnia (6%), nausea (6%), and anxiety (5%).

Pitolisant is contraindicated in patients with severe liver disease. The risk of QT prolongation may be greater in patients with liver or kidney disease. Pitolisant is not recommended in patients with end-stage kidney disease.

Full prescribing information is available online.

The company said pitolisant will be commercially available in the United States in the fourth quarter of this year.

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