Pitolisant Shows Positive Secondary Outcomes in Phase 2 Study

Exciting news for the Prader-Willi syndrome (PWS) community! Newly unveiled data from Harmony Biosciences' phase 2 study reveals promising impacts of pitolisant (Wakix) on PWS patients with excessive daytime sleepiness (EDS). Harmony Biosciences plans to kick off its phase 3 TEMPO study later this year, aiming to dive deeper into the potential of this treatment option.

The study's findings highlighted significant positive effects of pitolisant, particularly in the higher-dose group. In children aged 6 to 12, improvements were noted in various behavioral domains, including irritability and social withdrawal, painting a hopeful picture for the PWS community.

"We recognize the urgency for innovative treatments that help alleviate the profound unmet medical needs of individuals with PWS and their dedicated caregivers," said Dr. Kumar Budur, Harmony Biosciences' Chief Medical Officer.

Moreover, the study demonstrated noteworthy reductions in EDS severity and improvements in hyperphagia, further underlining the potential of pitolisant. With a safety profile in line with expectations, the study marks a crucial step forward in addressing the unmet needs of the PWS community.

The findings are set to inform the upcoming phase 3 study, emphasizing Harmony Biosciences' dedication to pioneering solutions for PWS. Stay tuned for more updates and click the button below to read the full announcement.

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