The Food and Drug Administration (FDA) approved Wakix (pitolisant; Harmony Biosciences) for the treatment of excessive daytime sleepiness (EDS) in adult patients with narcolepsy.
Wakix is a first-in-class, selective histamine-3 (H3) receptor antagonist/inverse agonist that works by increasing the synthesis and release of histamine, a wake-promoting neurotransmitter in the brain. It is the first treatment for narcolepsy that is not scheduled as a controlled substance.
The approval was based on data from 2 multicenter, double-blind, placebo-controlled studies (HARMONY 1 and HARMONY 1bis) that evaluated the efficacy of Wakix for the treatment of EDS in patients aged ≥18 years with narcolepsy (N=261). Patients were randomized to receive Wakix, placebo, or active control for a duration of 8 weeks with a 3-week dose titration phase followed by a 5-week stable dose phase; 75% to 80% of the patients reported a history of cataplexy.
Findings from both studies demonstrated that treatment with Wakix led to statistically significant improvements in EDS as measured by the Epworth Sleepiness Scale (ESS) score (primary end point; ESS is rated from 0 [would never doze] to 3 [high chance of dozing]), compared with placebo. At Week 8, the placebo subtracted difference ESS score in the HARMONY 1 study was -3.1 (95% CI: -5.73; -0.46) and -2.2 (95% CI: -4.17; -0.22) in the HARMONY 1bis study.
With regard to safety, the most common adverse reactions were insomnia, nausea, and anxiety. The Warnings and Precautions section includes information regarding the risk of QT interval prolongation with Wakix.
“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, MD “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.”
Wakix is expected to be commercially available in the fourth quarter of 2019. It will be supplied as 4.45mg and 17.8mg tablets.
For more information visit wakix.com.