The US Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for Harmony Biosciences LLC’s 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 (H?) 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 United States 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,” says John C. Jacobs, president and CEO at Harmony, in a release. “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.”

Jeffrey M. Dayno, MD, chief medical officer at Harmony, says, “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. 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 5 years. It also included safety data in over 1,500 patients across multiple patient populations.