Pushing the bounds of what science can do.

At Harmony Biosciences we are building a new kind of biopharmaceutical company, one that starts with gaining a deep understanding of the rare diseases we aim to treat—from the molecular level right on up to the day-to-day impact on the lives of people we try to help. Our goal is clear: to develop and commercialize affordable drugs that dramatically improve and extend the lives of patients with rare diseases who have long been underserved.

Our initial focus is treating central nervous system disorders, like narcolepsy, and our initial target is a receptor in the brain that controls the release of histamine. Histamine plays many roles in the brain, including making people feel awake and alert. The H3 receptor acts as the braking system for this histamine release, and it turns out, also helps regulate serotonin, acetylcholine, and dopamine. The more we look at H3, the more possibilities we see for treating a range of CNS disorders. First among them: cataplexy and excessive daytime sleepiness (EDS) in narcolepsy.

Advancing pitolisant, a novel drug.

The Harmony team is focusing its energy to bring to market the first drug in the U.S. that targets the H3 receptor: pitolisant, a selective histamine H3-receptor antagonist/inverse agonist for the potential treatment of cataplexy and excessive daytime sleepiness (EDS) in narcolepsy with and without cataplexy. Pitolisant boosts histamine release to improve daytime wakefulness. We are proud to be partnering with Dr. Jeanne-Marie Lecomte and Dr. Jean-Charles Schwartz, co-founders of French company Bioprojet SCR, to further develop, register, and file pitolisant with the Food and Drug Administration (FDA) and bring this product to market in the US. Pitolisant was approved in Europe in March 2016.