“We are inspired by the opportunity to bring a first-in-class treatment with a novel mechanism of action to patients in the U.S. living with narcolepsy, as there have been no new advancements to treat the most disruptive symptoms of this disorder in over a decade.”
Jeffrey Dayno, M.D., Chief Medical Officer
Currently, our work is focused on the development, registration, and marketing of our first product, pitolisant, a selective histamine 3 (H3) receptor antagonist/inverse agonist for the potential treatment of excessive daytime sleepiness and cataplexy in patients with narcolepsy. To date, no H3 receptor antagonist/inverse agonist has been approved by the U.S. Food & Drug Administration.
Pitolisant is an investigational medication in the U.S. 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 potent and highly 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 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.
The Pitolisant Expanded Access Clinical Evaluation (PEACE) program is an open-label Expanded Access Program (EAP) intended to provide access to pitolisant treatment for excessive daytime sleepiness in adult patients with narcolepsy and for cataplexy in adult patients with narcolepsy. 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: thepeacenarcolepsyprogram.com.
The company is currently evaluating the novel mechanism of action of pitolisant for broader applications in neurological disorders that may be mediated by the H3 receptor. This mechanism-based approach for pitolisant brings the potential for future clinical development in a variety of conditions beyond disorders of wakefulness.
Harmony is actively evaluating the potential addition of new compounds to its portfolio with a focus on orphan/rare neurological disorders.