FDA Approves Wakix for Cataplexy in Pediatric Narcolepsy Patients
The FDA has approved Harmony Biosciences' Wakix (pitolisant) for treating cataplexy in pediatric patients aged 6 years and older with narcolepsy, making it the first non-scheduled treatment for both pediatric and adult narcolepsy patients with or without cataplexy.
The Food and Drug Administration has expanded the approval of Wakix (pitolisant) to include treatment of cataplexy in pediatric patients aged 6 years and older with narcolepsy. Wakix is now the first-and-only FDA-approved non-scheduled treatment for pediatric and adult narcolepsy patients with or without cataplexy.
Wakix is an antagonist/inverse agonist of the histamine-3 receptor. With this approval, it is now indicated for the treatment of excessive daytime sleepiness (EDS) or cataplexy in patients aged 6 years and older with narcolepsy.
The expanded use for cataplexy in pediatric narcolepsy patients was supported by data from the double-blind, placebo-controlled phase 3 study that led to the approval of Wakix for EDS in these patients (ClinicalTrials.gov Identifier: NCT02611687). Results showed that among the 95 patients with a history of cataplexy, treatment with pitolisant significantly reduced the average number of cataplexy attacks per week compared with placebo (weekly rate of cataplexy [WRC]: 2.2 vs 5.6; WRC ratio, 0.39 [95% CI, 0.17-0.90]).
In pediatric patients, the most common adverse reactions reported were headache and insomnia. In the placebo-controlled phase of the clinical trial conducted in pediatric patients 6 years and older with narcolepsy with or without cataplexy, the most common adverse reactions (≥5% and greater than placebo) for Wakix were headache (19%) and insomnia (7%).
Wakix was approved by the FDA in August 2019 for the treatment of excessive daytime sleepiness in adult patients with narcolepsy, with approval expanded to include cataplexy in adult patients in October 2020. The FDA approved Wakix for the treatment of EDS in pediatric patients 6 years of age and older with narcolepsy in June 2024.
Wakix was granted orphan drug designation for the treatment of narcolepsy in 2010, and breakthrough therapy designation for the treatment of cataplexy in 2018. 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 (France). Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the US. The company continues to advance efforts toward obtaining pediatric exclusivity for Wakix, which, if granted, would add an additional six months of regulatory exclusivity for this growing franchise, with next-generation formulations having utility patents filed out to 2044.
Wakix prolongs the QT interval; avoid use of Wakix in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Wakix is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation and in patients with severe hepatic impairment.