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Harmony Biosciences releases a statement to the PWS Community

Response from Harmony Biosciences regarding Wakix (pitolisant)

A recent report from Scorpion Capital, a financial research group that specializes in 'activist short selling', makes several allegations about potential side effects of the drug Wakix (pitolisant), which is currently being evaluated in a clinical trial to treat excessive daytime sleepiness in Prader-Willi syndrome.  Harmony Biosciences, which produces Wakix and is conducting the PWS clinical trial, has responded with a statement to the community (see below).

We will continue to work with the PWS clinical experts and regulatory agencies to provide families with additional information regarding the potential risks and benefits of this drug, and any other potential treatment for PWS. 



Harmony Biosciences Community-Facing Statement


At Harmony Biosciences, we specialize in developing and delivering treatments for rare neurological diseases that others often overlook. We believe that where empathy and innovation meet, a better life can begin for people living with neurological diseases. Our team of experts from a wide variety of disciplines and experiences is driven by our shared conviction that innovative science translates into therapeutic possibilities for our patients, who are at the heart of everything we do.

WAKIX®, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness or cataplexy in adult patients with narcolepsy and has been commercially available in the U.S. since Q4 2019. WAKIX is safe, effective and enabled to be marketed around the globe. Its safety and efficacy have been evaluated by independent, peer-reviewed medical journals as well as regulatory bodies including the U.S. FDA, the E.M.A. and in other countries around the world.

Since Harmony’s founding in 2017, it has been our first core value to keep patients at the heart of everything we do. We treat all patient communities with empathy, compassion and commitment. We stand shoulder-to-shoulder with people living with rare diseases and the communities that support them.

In addition to high-touch patient support programs, our teams engage with patient advocacy groups and broad non-profit stakeholders, working to provide support for people living with rare neurological diseases. In 2019, Harmony Biosciences was selected as a recipient of Life Sciences Pennsylvania’s (LSPA) 2019 Patient Impact Award. The Patient Impact Award recognizes a medicine, therapy, device, company, or organization with operations in Pennsylvania that made a significant contribution to the quality of healthcare for patients.

We stand by the safety and efficacy of our product and our commitment to delivering it to patients in a way that is ethical and dignified. Ultimately, our goal at Harmony is as much about delivering, innovative, patient-centric solutions as it is about infusing the lives of those living with rare diseases with hope, dignity, and the confidence that comes from knowing they are no longer overlooked, they are no longer alone, and they are at the heart of everything we do.


Topics: Advocacy

Theresa Strong


Theresa V. Strong, Ph.D., received a B.S. from Rutgers University and a Ph.D. in Medical Genetics from the University of Alabama at Birmingham (UAB). After postdoctoral studies with Dr. Francis Collins at the University of Michigan, she joined the UAB faculty, leading a research lab focused on gene therapy for cancer and directing UAB’s Vector Production Facility. Theresa is one of the founding members of FPWR and has directed FPWR’s grant program since its inception. In 2016, she transitioned to a full-time position as Director of Research Programs at FPWR. She remains an Adjunct Professor in the Department of Genetics at UAB. She and her husband Jim have four children, including a son with PWS.