FDA Grants Breakthrough and Fast-Track Designations to Pitolisant for Treatment of Patients With Narcolepsy


The Food and Drug Administration (FDA) has granted fast-track and breakthrough therapy designations to the investigational therapy pitolisant (Harmony Biosciences, Plymouth Meeting, PA) for the treatment of excessive daytime sleepiness and cataplexy in patients with narcolepsy. The FDA grants the breakthrough therapy designations when there is preliminary clinical evidence that a therapy intended to treat a serious condition may provide substantial improvement in treatment compared to already available therapies. Fast-track designation is given when data demonstrate the potential to address an unmet medical need. These designations provide opportunities for frequent FDA interaction, possible rolling new drug application submission, and the potential for expedited review.

Pitolisant is the first selective histamine H3-receptor inverse agonist; it enhances the activity of histaminergic neurons in the brain, which function to improve a patient’s wakefulness and inhibit attacks of cataplexy. It has been developed by Bioprojet and has been available in Europe since 2016. If approved, pitolisant will be the first new therapy in over a decade for excessive daytime sleepiness and cataplexy in patients with narcolepsy in the US.     

Harmony also announced today that the pitolisant expanded access clinical evaluation (PEACE) program is open and patients are being enrolled; information on the PEACE program is available at www.thepeacenarcolepsyprogram.com.

“The expanded access program for pitolisant provides physicians in the U.S. with an opportunity to gain clinical experience with this first-in-class investigational product that works through the histaminergic system to improve wakefulness and inhibit attacks of cataplexy,” said Michael Thorpy, M.D., Professor of Neurology at Albert Einstein College of Medicine, Director of the Sleep-Wake Disorders Center at Montefiore Medical Center in New York, and Principal Investigator on the EAP for pitolisant. “I am pleased that Harmony Biosciences is making this program available while working on the NDA, as the data suggest that pitolisant could represent a new treatment option which is much needed for our patients with narcolepsy.”


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