Study evaluated the abuse potential of pitolisant compared to a schedule IV stimulant and placebo
Primary endpoint achieved – significantly lower maximum drug liking for pitolistant compared to a schedule IV stimulant
PLYMOUTH MEETING, PA, September 27, 2018 —Harmony Biosciences, LLC (Harmony) announced today that French pharmaceutical company Bioprojet SCR (Bioprojet), presented new human abuse potential (HAP) data on pitolisant at the 24th Congress of the European Sleep Research Society (ESRS) in Basel, Switzerland. Pitolisant is an investigational product in the U.S. that was studied in Europe and approved by the European Medicines Agency (EMA) in 2016 for the treatment of adult patients with narcolepsy with or without cataplexy. Harmony acquired exclusive rights in 2017 to develop and register pitolisant in the U.S.
“These data for pitolisant are exciting, as currently all FDA approved medications for the treatment of narcolepsy are scheduled, which indicates a potential for misuse and abuse,” said Harmony’s Chief Medical Officer, Jeffrey Dayno, M.D. “There is a significant unmet need for non-scheduled options that healthcare professionals can prescribe to treat patients living with narcolepsy.”
The HAP study of pitolisant evaluated the abuse potential of single doses of pitolisant compared to phentermine (Schedule IV stimulant) and placebo when administered to healthy, non-dependent, recreational stimulant users using a four period cross-over design. Pitolisant was given at a therapeutic dose of 40 mg and at a supratherapeutic dose of 240 mg while phentermine was given at a dose of 60 mg. The primary endpoint of the study was the peak maximum effect for Drug Liking (Emax). Key secondary endpoints included Overall Drug Liking and Take Drug Again. Topline results showed that both doses of pitolisant demonstrated statistically significant (P<0.0001) lower maximum drug liking scores compared to phentermine, and that the Emax scores for pitolisant were similar to placebo. This same pattern was seen on the secondary endpoints of Overall Drug Liking and Take Drug Again, with significantly lower scores for both doses of pitolisant compared to phentermine and scores for pitolisant that were similar to placebo.
Pitolisant is an investigational medication in the U.S. that is not 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 potent and highly selective histamine 3 (H3) receptor antagonist/inverse agonist; it enhances the activity of histaminergic neurons in the brain that function to improve a patient’s wakefulness and inhibit attacks of cataplexy. It was developed by Bioprojet who 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 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 (EDS) and cataplexy in patients with narcolepsy.
Narcolepsy is a rare, chronic, debilitating neurologic disorder of sleep-wake state instability that impacts up to 200,000 Americans and is primarily characterized by excessive daytime sleepiness, cataplexy, and other manifestations of REM sleep dysregulation, which intrude into wakefulness. In most patients, it is caused by the loss of hypocretin, a neuropeptide in the brain that supports sleep-wake state stability. This disorder affects men and women equally with typical symptom onset in adolescence or young adulthood; however, it 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.
Cataplexy is one of several symptoms of narcolepsy that represent elements of REM sleep state intruding into wakefulness, characterized by sudden temporary loss of muscle tone. Cataplexy can be subtle, such as drooping of eyelids, or severe, such as knee buckling or total body collapse. Often times, symptoms of cataplexy may go unrecognized because of the subtle nature of the symptoms in some patients, variability of how cataplexy is expressed, and/or lack of patient complaints or physician recognition of the symptoms as manifestations of cataplexy. This symptom of narcolepsy can often cause significant impact on a person’s ability to carry out normal daily functions. Up to two-thirds of all narcolepsy patients have cataplexy (known as Type 1 narcolepsy); cataplexy is one of the most debilitating symptoms of this chronic, rare neurologic disorder.
Harmony Biosciences, LLC
Harmony Biosciences, LLC, is a private biopharmaceutical company headquartered in Plymouth Meeting, PA. The company was established in October 2017 by global biopharmaceutical incubator and investment firm, Paragon Biosciences, LLC, 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 living with narcolepsy. Harmony Biosciences is committed to advancing the understanding of narcolepsy and providing information and resources to individuals who live with narcolepsy and healthcare professionals who treat patients with this disorder. 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 about 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
Harmony Biosciences, LLC Media Contact: