Pitolisant Granted Breakthrough Therapy, Fast Track Designations by FDA


The therapy is a first of the histamine H3 receptor-antagonist/inverse agonist class—working by enhancing the histaminergic neurons in the brain.

The US Food and Drug Administration (FDA) has granted Harmony Biosciences a Breakthrough Therapy designation for pitolisant, an investigational therapy for the treatment of cataplexy in patients with narcolepsy.

The therapy was also granted a Fast Track designation as well, as there are many unmet needs in the realm of narcolepsy. Misdiagnosis of the condition is common—roughly 25% of those with it have been diagnosed—while it impacts approximately 1 in every 2000 Americans.

“We are very pleased that the FDA has granted pitolisant Breakthrough Therapy and Fast Track designations and believe it reflects the Agency’s interest in this investigational product to potentially address an important unmet medical need for patients with narcolepsy,” said Jeffrey Dayno, MD, the chief medical officer at Harmony, said in a statement. “We look forward to working with the FDA throughout the submission and review of an NDA for this first-in-class molecule with a novel mechanism of action to treat adult patients with narcolepsy with or without cataplexy.”

Harmony Biosciences also announced the beginnings of enrollment for the Pitolisant Expanded Access Clinical Evaluation (PEACE) program. As part of the Expanded Access Program (EAP), the open-label evaluation will provide the therapy to adults in the US with excessive daytime sleepiness associated with narcolepsy, with or without cataplexy as a symptom.

Michael Thorpy, MD, a professor of neurology at Albert Einstein College of Medicine, and the director of the Sleep-Wake Disorders Center at Montefiore Medical Center in New York City, is serving as the principal investigator on the EAP for pitolisant. He said in a statement that the program will give physicians in the US an opportunity to gain clinical expertise with a “first-in-class investigational product that works through the histaminergic system to improve wakefulness and inhibit attacks of cataplexy.”

“I am pleased that Harmony Biosciences is making this program available while working on the [New Drug Application], as the data suggest that pitolisant could represent a new treatment option which is much needed for our patients with narcolepsy,” he added.

The therapy is the first of the histamine H3 receptor-antagonist/inverse agonist class—working by enhancing the histaminergic neurons in the brain. It received approval by the European Medicines Agency in 2016. The goal for approval in the US is 2019.

“Receiving Breakthrough and Fast Track designations for pitolisant from FDA is welcome news as narcolepsy patients face many challenges,” said Bob Repella, Harmony’s CEO. “As stated in FDA’s Voice of the Patient report on Narcolepsy, it is a debilitating disease that can severely affect a patient’s day-to-day functioning and have a devastating impact on their lives.”

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