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Harmony Biosciences Presented Data On Pitolisant

September 12th, 2018

Harmony Biosciences, LLC (Harmony) presented data on its investigational product, pitolisant, which has been studied for the treatment of both excessive daytime sleepiness (EDS) and cataplexy in adult patients with narcolepsy, at the upcoming 7th International Symposium on Narcolepsy.

Data presented included:

  • Oral Presentation: An Open-Label, Naturalistic Study to Assess the Long-Term Safety of Pitolisant in Adult Patients with Narcolepsy With or Without Cataplexy: Results from the Harmony III Study Five Year Extension
    • Harmony III study evaluated the long-term (up to 5 years) safety and tolerability of pitolisant in an open-label naturalistic study that included patients who were both naïve and experienced on pitolisant therapy.   This presentation included findings from the French cohort from this trial that extended out to 5 years. Long-term safety/tolerability data was presented, along with open-label data on the durability of effect of pitolisant over time on EDS and symptoms of REM dysregulation such as cataplexy, hypnagogic hallucinations, and sleep paralysis.
  • Poster Presentation: Pitolisant Treatment of Excessive Daytime Sleepiness and Cataplexy in Patients with Narcolepsy: an Open-Label, Expanded Access Program in the United States (PEACE)
    • This presentation described the design and methodology of the program, as well as report out initial data, including patient demographics, baseline characteristics and safety, and tolerability results.

The Pitolisant Expanded Access Clinical Evaluation (PEACE) program is an open-label Expanded Access Program (EAP) intended to provide treatment with pitolisant to adult patients in the US with excessive daytime sleepiness associated with narcolepsy with or without cataplexy. It is open to all qualified healthcare professionals who wish to participate and interested patients who meet the inclusion/exclusion criteria of the protocol. For more information on the PEACE program, visit:

About Pitolisant

Pitolisant is an investigational medication in the US that is not currently 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 is the first selective histamine 3 (H3) receptor antagonist/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 was developed by Bioprojet which has marketed the product in Europe since its approval by the European Medicines Agency in 2016. The receipt of Breakthrough Therapy and Fast Track designations for pitolisant afforded Harmony the opportunity to request a rolling NDA submission to the FDA, which was granted. Harmony’s goal is to obtain FDA approval to market this new medication in the US in 2019. If approved, pitolisant would represent the first new therapy in the US in over a decade for the treatment of excessive daytime sleepiness (EDS) in patients with narcolepsy, and in fifteen years for the treatment of cataplexy in patients with narcolepsy.

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Date Created: September 12th, 2018
Last Updated: December 8th, 2020