Harmony Biosciences Secures U.S. FDA Approval for WAKIX® (pitolisant) to Treat Cataplexy in Paediatric Narcolepsy
Wednesday, February 18, 2026
Harmony Biosciences has received approval from the U.S. Food and Drug Administration for its supplemental new drug application (sNDA) for WAKIX® (pitolisant) tablets to treat cataplexy in paediatric patients aged six years and older with narcolepsy.
With this decision, WAKIX becomes the first and only FDA-approved non-scheduled treatment for both paediatric and adult patients with narcolepsy, with or without cataplexy. Clinicians can now prescribe the medicine to manage excessive daytime sleepiness (EDS), cataplexy, or both, in patients aged six years and above.
WAKIX was first approved in August 2019 for the treatment of EDS in adults with narcolepsy. The approval was extended in October 2020 to include cataplexy in adults. In June 2024, the FDA also approved WAKIX for EDS in paediatric patients aged six years and older with narcolepsy.
The company is continuing efforts to obtain paediatric exclusivity for WAKIX, which, if granted, would provide an additional six months of regulatory exclusivity. It is also progressing development of next-generation formulations of pitolisant for additional indications, with utility patents filed through to 2044.
WAKIX is a first-in-class medicine approved for the treatment of EDS or cataplexy in patients aged six years and older with narcolepsy. It received orphan drug designation in 2010 and breakthrough therapy designation in 2018 for the treatment of cataplexy. The medicine acts as a selective histamine 3 (H₃) receptor antagonist/inverse agonist. While its exact mechanism of action is not fully understood, it is believed to increase the synthesis and release of histamine, a neurotransmitter that promotes wakefulness.
Pitolisant was designed and developed by Bioprojet. Harmony Biosciences holds an exclusive licence from Bioprojet to develop, manufacture and commercialise pitolisant in the United States.
Source: businesswire.com
