Pitolisant New Drug Application Granted Priority Review

Pitolisant (Harmony Biosciences; Plymouth Meeting, PA), an investigational drug being studied for the treatment of excessive daytime sleepiness and/or cataplexy in patients with narcolepsy, has been granted priority review of the new drug application for it by the Food and Drug Administration (FDA). A first-in-class small molecule, pitolisant is thought to act through an antagonist/inverse agonist effect on the histamine 3 (H3) receptor. The priority review designation by the FDA indicates that pitolisant could represent a significant improvement in safety or efficacy compared to existing treatments for patients with narcolepsy. 

Long-term data showed that patients with narcolepsy treated with pitolisant had improvements in EDS measured with the Epworth Sleepiness Scale (ESS and a favorable safety profile with no evidence of tolerability or withdrawal symptoms seen when patients stopped using pitolisant. 

 “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 excessive daytime sleepiness 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.”