PLYMOUTH MEETING, Pa. - Harmony Biosciences Holdings , Inc. (NASDAQ: NASDAQ:HRMY) has received priority review designation from the U.S. Food and Drug Administration (FDA) for its supplemental New Drug Application (sNDA) for WAKIX® (pitolisant) tablets. This application seeks to expand the use of WAKIX® for treating excessive daytime sleepiness (EDS) or cataplexy in pediatric patients aged 6 years and older with narcolepsy. The FDA has set a target action date of June 21, 2024, under the Prescription Drug User Fee Act (PDUFA).
The company's Chief Executive Officer, Jeffrey M. Dayno, M.D., expressed that the FDA's decision to grant priority review underscores the urgent need for new treatments for children with this condition and accelerates the review process. Harmony Biosciences is looking to offer a new treatment option for pediatric patients, pending FDA approval.
The priority review is based on the results of a Phase 3 study, which was a multicenter, randomized, placebo-controlled trial evaluating the safety and efficacy of pitolisant in pediatric patients with narcolepsy. Following the study, the European Medicines Agency approved the extension of pitolisant's indication to include treatment for children aged 6 and older with narcolepsy, with or without cataplexy.
Narcolepsy is a rare, chronic neurological disorder that affects sleep-wake cycles, with approximately 170,000 Americans diagnosed with the condition. It is characterized by EDS and cataplexy, among other symptoms. WAKIX® operates as a selective histamine 3 (H₃) receptor antagonist/inverse agonist, with its efficacy potentially mediated through increased synthesis and release of histamine, a wake-promoting neurotransmitter.
Currently, WAKIX® is FDA-approved for the treatment of EDS or cataplexy in adult patients with narcolepsy and has been available in the U.S. since the fourth quarter of 2019. The drug has received orphan drug designation for narcolepsy treatment in 2010 and breakthrough therapy designation for cataplexy treatment in 2018.
The safety and effectiveness of WAKIX® for pediatric use are under review by the FDA, and the company eagerly anticipates the possibility of providing an additional therapeutic option for children suffering from narcolepsy. This news is based on a press release statement from Harmony Biosciences.
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