PLYMOUTH MEETING, PA, May 21, 2018 — Harmony Biosciences, LLC (Harmony), today announced that it has received Breakthrough Therapy and Fast Track designations for its investigational product, pitolisant, from the U.S. Food and Drug Administration (FDA). Breakthrough Therapy designation has been granted for pitolisant for the treatment of cataplexy in patients with narcolepsy and is received when a potential new therapy is intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies. Fast Track designation has been granted for the investigation of pitolisant for the treatment of excessive daytime sleepiness in patients with narcolepsy and the treatment of cataplexy in patients with narcolepsy and is received when a potential new therapy is intended to treat a serious condition and data demonstrate the potential to address an unmet medical need. These designations signal FDA’s interest in the development program for pitolisant and provide opportunities for frequent interaction with the Agency, the ability to request a rolling new drug application (NDA) submission, as well as the potential for expedited review.
“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 Harmony’s Chief Medical Officer, Jeffrey Dayno, M.D. “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.”
“Receiving Breakthrough and Fast Track designations for pitolisant from 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.”
Harmony also announced today that the Pitolisant Expanded Access Clinical Evaluation (PEACE) program is open and patients are being enrolled. This is an open-label Expanded Access Program (EAP) intended to provide treatment with pitolisant to adult patients in the U.S. 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 www.thepeacenarcolepsyprogram.com.
“The expanded access program for pitolisant provides physicians in the U.S. 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,” said Michael Thorpy, M.D., 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.”
Pitolisant is an investigational medication in the U.S. that is not currently approved by the FDA. It received orphan designation by the FDA for the treatment of narcolepsy. 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. It has been 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 allows Harmony to request a rolling submission from the FDA with the goal of obtaining approval to market this new medication in the U.S. in 2019. If approved, pitolisant would represent the first new therapy in the U.S. in over a decade for the treatment of both excessive daytime sleepiness and cataplexy in patients with narcolepsy.
Narcolepsy is a rare, chronic, debilitating neurologic disease of sleep-wake state instability that impacts approximately one in 2,000 Americans. It is characterized by excessive daytime sleepiness (EDS) and elements of rapid eye movement (REM) sleep (e.g., cataplexy, sleep paralysis, hallucinations) intruding into wakefulness. In most patients, it is caused by the loss of hypocretin, a neuropeptide in the brain that supports sleep-wake state stability. The disease affects men and women equally with typical symptom onset in adolescence or young adulthood; however, the disease can take up to a decade to be properly diagnosed. Narcolepsy can cause significant burden on patients and their families, affecting their ability to perform routine tasks, limit achievement at school and work, impact social relationships and cause impairment in overall quality of life.
Harmony Biosciences, LLC
Harmony Biosciences, LLC, is a privately-owned biopharmaceutical company headquartered in Plymouth Meeting, PA. The company was established in October 2017, with a vision to provide novel treatment options for people living with rare and orphan diseases with an emphasis on central nervous system disorders, starting with patients who live with narcolepsy. Harmony Biosciences is committed to advancing the understanding of narcolepsy and providing information and resources to individuals who live with, and healthcare professionals who treat patients with narcolepsy. On February 28, 2018, National Rare Disease Awareness Day, Harmony Biosciences launched Know Narcolepsy™, an educational campaign to help increase understanding of the disease and provide a new resource for the community. KnowNarcolepsy.com includes information on narcolepsy and cataplexy as well as powerful patient stories about living with the disease. Harmony Biosciences received the 2017 Deal of the Year Award from Life Sciences PA. For more information on Harmony Biosciences, visit www.harmonybiosciences.com.