Rare Disease Report

Pitolisant Receives FDA Breakthrough Therapy and Fast Track Designations

MAY 21, 2018
Krista Rossi
This morning, Harmony Biosciences, LLC announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy and Fast Track designations to its product, pitolisant, for the treatment of excessive daytime sleepiness and cataplexy in patients with narcolepsy.

Harmony's Chief Medical Officer, Jeffrey Dayno, MD, expressed his excitement over the drug’s designations in a recent statement. "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. 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."

Pitolisant is the first selective histamine 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.

"Receiving Breakthrough and Fast Track designations for pitolisant from the FDA is welcome news as narcolepsy patients face many challenges," said Harmony's CEO, Bob Repella. "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."

Narcolepsy is a debilitating, chronic, neurological disease that is characterized by a sleep-wake state. Excessive daytime sleepiness (EDS) and elements of rapid eye movement (REM) sleep (e.g., cataplexy, sleep paralysis, hallucinations) intruding into wakefulness are common symptoms. The loss of hypocretin is most common factor in narcolepsy patients.

"The expanded access program for pitolisant provides physicians in the US with an opportunity to gain clinical experience with this first-in-class investigational product that works through the histaminergic system to improve wakefulness and inhibit attacks of cataplexy," commented Michael Thorpy, MD, Professor of Neurology at Albert Einstein College of Medicine, Director of the Sleep-Wake Disorders Center at Montefiore Medical Center in New York, and Principal Investigator on the EAP for pitolisant. "I am pleased that Harmony Biosciences is making this program available while working on the NDA, as the data suggest that pitolisant could represent a new treatment option which is much needed for our patients with narcolepsy."

In addition, Harmony announced the commencement of its Pitolisant Expanded Access Clinical Evaluation (PEACE) program, which patients are currently being enrolled in. As an open-label Expanded Access Program (EAP), it aims at providing treatment with pitolisant to adult patients in the US who experience excessive daytime sleepiness associated with narcolepsy or without cataplexy. All patients who meet the inclusion/exclusion criteria of the protocol and all qualified healthcare professionals are eligible to participate.

"Receiving Breakthrough and Fast Track designations for pitolisant from the FDA is welcome news as narcolepsy patients face many challenges," emphasized Harmony's CEO, Bob Repella. "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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