Written by Marcin Miszkowski
What’s the news?
Trials of the use of Pitolisant to ease excessive daytime sleepiness in people with PWS have shown some positive early results after testing of a group aged 6-65. The eleven-week placebo-controlled clinical trial showed small but meaningful top-line data, with further results expected by the end of the year. It is hoped that some of this data may shed light on secondary goals of the trial, including behavioural changes, cognitive function and excessive hunger.
What’s the science?
An 11-week trial conducted by Harmony, pitolisant’s manufacturer, saw 65 patients randomly administered a course of high-dose or low-dose pitolisant, or a matching placebo for the duration of the trial. Courses were triturated (increased) up to the final dose for the first three weeks, with the final dose being administered for eight-weeks. Dose strength varied depending on if the participants were paediatric, adolescent or adult. Results were measured by caregivers using the Epworth Sleepiness Scale. Daytime sleepiness scores dropped notably in adults and children, but showed no meaningful results from the adolescent group. Overall, 70% of participants from the high-dose group, 56% of the low-does, and 53% of the placebo-controlled group showed a significant point difference in their sleepiness scores. About a quarter of the pitolisant group reported side effects consistent with the pitolisant profile.
What does this mean for people with PWS?
The Foundation for Prader-Willi Research and Prader-Willi Syndrome Association USA reported in a joint statement that Harmony Biosciences, pitolisant’s developer, are intending to continue with trials into furthering treatment options for people with PWS. This is encouraging news for a community with low treatment options.