Pitolisant approved for treatment of excessive daytime sleepiness in adult patients with narcolepsy

Harmony Biosciences has announced that the U.S. Food and Drug Administration has approved WAKIX® (pitolisant) for the treatment of excessive daytime sleepiness (EDS) in adult patients with narcolepsy.  


WAKIX, a first-in-class medication, is a selective histamine 3 (H₃) receptor antagonist/inverse agonist that works through a novel mechanism of action to increase the synthesis and release of histamine, a wake-promoting neurotransmitter in the brain. WAKIX is administered orally once daily in the morning upon wakening.

Excessive daytime sleepiness is one of many challenges people with PWS face and this approval will provide adults with PWS who experience daytime sleepiness with narcolepsy a new treatment avenue. At this time, pitolisant has only been approved for adults with narcolepsy, however, FPWR is working closely with Harmony to help them evaluate pitolisant in a clinical trial specific for people with PWS.

A Message from Harmony Biosciences:

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. 

Topics: News

Susan Hedstrom

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Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the natural history of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she hosted her first One SMALL Step walk in 2010 and began the development of the One SMALL Step walk program which now raises over $1.5 million a year for PWS research.

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