FDA to Review Pitolisant for Narcolepsy Treatment

Harmony Biosciences announced that the Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for pitolisant for the treatment of excessive daytime sleepiness (EDS) and/or cataplexy in adults with narcolepsy.

Pitolisant is an investigational, first-in-class molecule that acts as a potent and highly selective histamine 3 (H₃) receptor antagonist/inverse agonist. The NDA submission is supported by data from a clinical development program that included over 300 patients with narcolepsy, as well as safety data in over 1500 patients. Pitolisant was previously granted Orphan Drug, Fast Track, and Breakthrough Therapy designations by the FDA.  

“Pitolisant offers a novel approach to the treatment of both EDS and cataplexy in patients with narcolepsy, for which there have been no new treatment options in over 15 years,” said Jeffrey M. Dayno, MD, Chief Medical Officer at Harmony. “We look forward to working with the FDA during its review of the pitolisant NDA, with our hope of being able to offer this new treatment option to help address an important unmet medical need for people living with narcolepsy.”

For more information visit HarmonyBiosciences.com.