HARMONY BIOSCIENCES ANNOUNCES FILE ACCEPTANCE OF ITS NEW DRUG APPLICATION FOR PITOLISANT
FDA GRANTS PRIORITY REVIEW OF THE PITOLISANT NDA
PLYMOUTH MEETING, PA, February 12, 2019 — Harmony Biosciences, LLC (Harmony) announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for its investigational product, pitolisant, and has granted Priority Review for this NDA. Pitolisant is a first-in-class molecule with a novel mechanism of action; it is a potent and highly selective histamine 3 (H3) receptor antagonist/inverse agonist for the potential treatment of excessive daytime sleepiness (EDS) and/or cataplexy in adult patients with narcolepsy. A Priority Review designation by the FDA indicates that, if approved, pitolisant would provide a significant improvement in the safety or effectiveness of the treatment of EDS and/or cataplexy in adult patients with narcolepsy when compared to existing treatments. Harmony’s goal is to obtain FDA approval to market pitolisant in the U.S. in 2019.
« The impact of narcolepsy can be significant, and severely disruptive to everyday life for up to 200,000 Americans living with this disorder, » said John C. Jacobs, President and CEO at Harmony. « This is an important step for patients and for our company, whose mission is to develop novel treatment options for people living with rare and orphan diseases. »
« 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, M.D., 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. »
The NDA submission is based on results from the clinical development program in narcolepsy which included over 300 patients, some of whom were treated for up to five years. It also included safety data in over 1500 patients across multiple patient populations.
Pitolisant is an investigational medication in the U.S. that is not approved by the FDA. It was granted orphan designation for the treatment of narcolepsy, Fast Track designation for the treatment of excessive daytime sleepiness (EDS) and cataplexy in patients with narcolepsy, and Breakthrough Therapy designation for the treatment of cataplexy in patients with narcolepsy. Pitolisant, a first-in-class medication, is a potent and highly selective histamine 3 (H3) receptor antagonist/inverse agonist; it enhances the activity of histaminergic neurons in the brain that function to improve a patient’s