FDA Approves Pitolisant for Treatment of Narcolepsy

Maria Picone
Maria Picone

Yesterday, Harmony Biosciences announced FDA Approval Of WAKIX (pitolisant), A First-In-Class Medication For The Treatment Of Excessive Daytime Sleepiness In Adult Patients With Narcolepsy. Read their press release here:


This is big news for the community because pitolisant is the first and only non-scheduled treatment approved for patients with narcolepsy in the U.S. We are also excited to announce Harmony’s plans to evaluate pitolisant as a treatment for the symptoms of Prader-Willi Syndrome. Following is an announcement received from Harmony regarding this effort. We applaud their hard work to improve the lives of patients living with rare disease.

As you may have heard, on August 14, Harmony Biosciences, LLC (Harmony) announced that the U.S. Food and Drug Administration approved WAKIX® (pitolisant) for the treatment of excessive daytime sleepiness (EDS) in adult patients with narcolepsy. Based on questions we have been receiving from the Prader-Willi Syndrome (PWS) community, we would like to provide you with an update related to our clinical development program for PWS.

Harmony is very interested in evaluating pitolisant in people living with PWS. Our company is currently pursuing a clinical development program, for which we have engaged with the FDA, with the intent of characterizing the safety, efficacy and appropriate dosing of pitolisant in treating some of the symptoms experienced by patients with PWS.

At Harmony, it is inherent in our culture to keep patients at the heart of everything we do, including addressing unmet medical needs and developing treatments that may help people living with rare diseases. We understand there is a great need and sense of urgency for new treatments in the PWS community. Harmony is committed to engaging with the PWS community as we explore the potential for pitolisant in patients with PWS.