FDA Approves WAKIX for Cataplexy Treatment in Pediatric Narcolepsy Patients
The FDA approved Harmony Biosciences' supplemental new drug application for WAKIX (pitolisant) to treat cataplexy in pediatric patients 6 years and older with narcolepsy, making it the first non-scheduled treatment for both pediatric and adult narcolepsy patients with or without cataplexy.
Harmony Biosciences announced that the U.S. Food and Drug Administration has approved its supplemental new drug application for WAKIX (pitolisant) tablets for the treatment of cataplexy in pediatric patients 6 years of age 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.
Cataplexy is a narcolepsy symptom characterized by sudden muscle weakness. WAKIX, or pitolisant, had previously been approved for adults with or without cataplexy, and for pediatric patients without cataplexy.
With this approval, clinicians now have the option to prescribe WAKIX to treat excessive daytime sleepiness, cataplexy, or both, in patients 6 years and older with narcolepsy.
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 excessive daytime sleepiness in pediatric patients 6 years of age and older with narcolepsy in June 2024.
The company's Chief Executive Officer stated that WAKIX is the first-and-only FDA-approved treatment option for both pediatric and adult narcolepsy patients with or without cataplexy that is not scheduled as a controlled substance, an important distinction that supports its clinical utility. The company continues to advance its efforts toward obtaining pediatric exclusivity for WAKIX, which, if granted, would add an additional six months of regulatory exclusivity for this growing franchise.
The company's sights are now set on further growing, extending and expanding the value of pitolisant through additional indications with next-generation formulations, which have utility patents filed out to 2044.
WAKIX, a first-in-class medication, 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 United States.
WAKIX was granted orphan drug designation for the treatment of narcolepsy in 2010, and breakthrough therapy designation for the treatment of cataplexy in 2018.
Shares of Harmony were up 3.9% to $37.42 in Tuesday afternoon trading.