MAY 2019 – The case series data from 3 children trialling pitolisant has been published. It was noted that the treatment was well tolerated and in addition to the previously reported positive effects on cognition (mental clarity and processing speed), pitolisant had the additional benefit of normalising the children’s relationship to food. You can read more details about the dosing and any side effects in The Chion Foundation’s April press release. The Chion Foundation have also released the short film below about patient experience with pitolisant.
Pitolisant is a histamine 3 receptor inverse agonist which was newly approved in the European Union for the treatment of narcolepsy and cataplexy. It was identified as a potential treatment for PWS by Lara Pullen PhD, co-founder of The Chion Foundation, when she noted that neurotransmitters which are dysregulated in individuals with PWS are those that are regulated by the histamine 3 receptor (H3R). The H3R regulates sleep/wake states, hunger, cognition, and movement, which are all affected in PWS. The Chion Foundation partnered with TREND Community to help families import pitolisant under the FDA’s personal importation program and to collect trial data from these families.
Dosing data from 9 children treated with pitolisant was first presented at the FPWR annual research symposium in 2018. In 9 children, benefits were observed at a higher dose than is usual for treating narcolepsy, although a tenth child had to stop treatment due to side effects. Detailed data was presented for the 3 children in the case series above and it was explained that although they had previously been frequently sleepy during the day, as is typical of many children with PWS, they had not presented with typical adult symptoms of narcolepsy. This led to the conclusion that individuals with PWS, with and without a diagnosis of narcolepsy, may benefit from pitolisant.
Thanks to a partnership between The Chion Foundation and Harmony Biosciences, in 2018 Pitolisant became available for the treatment of narcolepsy in the USA under an expanded access program and then received FDA Breakthrough Therapy Designation.