The Chion Foundation, an organization committed to improving the lives of individuals affected by Prader-Willi Syndrome (PWS) and other rare diseases, has partnered with Trend Community, an innovative patient experience and engagement platform, to launch the Pitolisant Health Initiative to explore the potential of pitolisant (sold under the brand name of Wakix™) to address the many unmet needs of patients with PWS. PWS is a rare, complex disease characterized by hunger, disturbances in the sleep/wake cycle, excessive daytime sleepiness, low muscle tone, and decreased cognition. Pitolisant is an inverse agonist of the histamine h3 receptor recently approved in Europe as a treatment for the rare sleep disorder of narcolepsy with or without cataplexy.
While pitolisant is currently only available in Europe, the Chion Foundation and TREND Community have worked with the US Food and Drug Administration (FDA) to help individuals personally import pitolisant from Europe based upon the FDA’s discretion. These families are documenting their experience with pitolisant on TREND Community via journaling and survey questions.
Thus far, the Chion Foundation has identified seven US families affected by PWS who have been prescribed and are now taking pitolisant via the FDA’s personal importation program. Caregiver reported data suggest that pitolisant is well-tolerated and has neurological benefits that extend beyond wakefulness to include mental clarity, increased processing speed, improved attention, and improved muscle tone. Early patient experience with pitolisant is encouraging.
Immunologist and co-founder of Chion Foundation, Lara C. Pullen, PhD identified pitolisant as an ideal drug to be repurposed to treat many of PWS’ clinical manifestations. The histamine h3 receptor is located in the brain and gut, and is known to regulate not only sleep/wake states, but also hunger, cognition, and movement, all of which are debilitating problems for patients with PWS.
Today Harmony Biosciences announced its licensing agreement with Bioprojet Pharma to bring Wakix to market in the United States (read press release). The Chion Foundation and Trend Community intend to continue its collection of patient experience data.
About Chion Foundation: https://www.chionfoundation.org/
About Trend Community: https://trend.community/
About FDA Personal Importation Program: https://www.fda.gov/ForIndustry/ImportProgram/ImportBasics/ucm432661.htm
About Harmony Biosciences: https://harmonybiosci.squarespace.com/