Harmony Biosciences, a pharmaceutical company focused on developing therapies for rare neurological diseases, has announced its plans to conduct a Phase 3 registrational study of pitolisant in individuals with Prader-Willi syndrome (PWS) who are experiencing excessive daytime sleepiness (EDS). This study will include children, adolescents, and adults with PWS. The decision to proceed with the study follows a successful End-of-Phase 2 meeting with the FDA. Harmony aims to further investigate the effectiveness and safety of pitolisant as a potential treatment for EDS in individuals with PWS, as there is currently no FDA-approved treatment available for this condition.
Prader-Willi syndrome is a genetic neurological disorder characterized by symptoms that arise from dysfunction in the hypothalamus. These symptoms include excessive daytime sleepiness and hyperphagia, which is an intense sensation of hunger. More than half of patients with PWS experience excessive daytime sleepiness. Harmony's pitolisant, marketed under the name WAKIX®, is already approved by the FDA for treating excessive daytime sleepiness or cataplexy in adult patients with narcolepsy but has not been approved for