Harmony Biosciences Holdings Inc announced that the US Food and Drug Administration (FDA) granted priority review for its supplemental New Drug Application (sNDA) for Wakix (pitolisant) tablets for the treatment of excessive daytime sleepiness or cataplexy in pediatric patients 6 years of age and older with narcolepsy.
The FDA has set a Prescription Drug User Fee Act, or target action date, of June 21.
“The FDA’s decision highlights the pressing need for new treatment options for children living with narcolepsy and expedites the timeline for its decision,” says Jeffrey M. Dayno, MD, president and CEO at Harmony Biosciences, in a release. “We are committed to expanding the utility of pitolisant for patients with high unmet medical needs and look forward to collaborating with the FDA to potentially introduce a new, non-scheduled treatment option for children with narcolepsy.”
The FDA accepted the sNDA and granted priority review based on a phase 3 multicenter, randomized, placebo-controlled study conducted by Bioprojet that evaluated the safety and efficacy of pitolisant in pediatric patients age 6 to under 18 years with narcolepsy with or without cataplexy.
Based on the results of this study, Bioprojet received approval from the European Medicines Agency last year extending the indication for pitolisant to include the treatment of narcolepsy in children aged 6 years and older, with or without cataplexy.
Wakix, a first-in-class medication, is approved by the FDA for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy and has been commercially available in the United States since late 2019.
It was granted orphan drug designation for the treatment of narcolepsy in 2010 and breakthrough therapy designation for the treatment of cataplexy in 2018.
