Pitolisant Expanded Access Program

Pitolisant is now available in the United States. Adults who have a diagnosis of narcolepsy with or without cataplexy should discuss this option with their sleep specialist. Harmony Biosciences’ expanded access program (EAP) will provide access to treatment with pitolisant while a U.S. New Drug Application (NDA) is being prepared and submitted for review for marketing approval. Patients should direct their physicians to the EAP described at: https://www.clinicaltrials.gov/ct2/show/NCT03433131?term=pitolisant&rank=7

Pitolisant is available in the European Union (E.U.) under the brand name of Wakix®. In the E.U. it is approved for adults as a treatment of narcolepsy with or without cataplexy. Pitolisant is a potent, orally active histamine H3-receptor antagonist/inverse agonist. It interacts with these histamine auto-receptors and is thus able to enhance the activity of brain histaminergic neurons. The histaminergic neurons constitute a major arousal system with widespread projections throughout the whole brain. Pitolisant also modulates various neurotransmitter systems, increasing acetylcholine, noradrenaline and dopamine release in the brain.

Narcolepsy (with or without cataplexy) is a chronic condition. Studies have demonstrated that pitolisant improves the level and duration of wakefulness and daytime alertness in patient with narcolepsy with or without cataplexy. The studies used the Maintenance of Wakefulness Test (MWT) and Sustained Attention to Response Task (SART) to document these effects.

For more details, please see the E.U. package insert, available here: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/002616/WC500204746.pdf