Pitolisant Program USA
The Pitolisant Program has two main goals:
Determine if pitolisant can relieve the burden of sleep disorders in PWS and possibly have positive effects on some of the other characteristics of this disease, and
If pitolisant is beneficial for patients with PWS, who have disordered sleep, work to provide access in the United States.
Pitolisant (Wakix) was approved by the European Medicines Agency in fall 2015 (http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/002616/human_med_001955.jsp&mid=WC0b01ac058001d124) for the treatment of narcolepsy. The drug is currently available in parts of Europe.
Pitolisant represents the first of a new class of drugs. This means that there is very little clinical experience with the drug. Early evidence suggests, however, that it has an excellent safety profile.
Pitolisant targets the histamine 3 receptor (H3R) and is an inverse agonist. The H3R is found primarily in the nervous system and gastrointestinal tract. It has many functions throughout the body and its role in health and disease is under active investigation. Thus far, scientists know that the H3R regulates many neurotransmitters (brain chemicals) and plays a role in the regulation of sleep and hunger. Scientists learn and publish more about the H3R every month.
Recently Holger Stark, PhD of the Heinrich Heine University of Dusseldorf in Germany presented his research on pitolisant and the H3R at the International Prader-Willi Syndrome Organization meeting in Toronto. Dr. Stark is an international leader in research on the H3R and his presentation raised the possibility that pitolisant may benefit individuals with PWS.
We at the Chion Foundation hypothesize that pitolisant may be uniquely positioned to meet the needs of patients with PWS. We propose that it will normalize sleep, behavior, and cognition. We are also eager to explore whether or not it will relieve other burdens of the complex syndrome.
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