Blue backgound with Harmony Bioscienes label for pitolisant as treatment for Prader-Willi Syndrome

Pitolisant Receives Orphan Drug Designation

Pitolisant from Harmony Biosciences Receives Orphan Drug Status from FDA for Treatment of PWS

The FDA has granted Orphan Drug status to pitolisant, the trial drug from Harmony Biosciences to treat excessive daytime sleepiness (EDA) and behavioral disturbances. The designation shows that the FDA considers pitolisant to be a promising treatment for people with PWS. 

Orphan drug designation is given to those treatments intended for rare conditions and provide the company with marketing and development rights, tax credits, and fee waivers. 

“The designation marks an important step forward in our PWS development program and we are eager to continue working with the FDA and the broader community of PWS patients and caregivers to address their high unmet medical needs,” said Kumar Budur, M.D., M.S., Chief Medical Officer at Harmony Biosciences.

Pitolisant has shown to have positive effects on EDS, hyperphagia, and behavior in individuals with PWS, including irritability and social withdrawal. Harmony is looking towards the upcoming Phase 3 registration TEMPO study to assess the safety and efficacy of pitolisant. 

This new designation is truly exciting news for the PWS community and the growing possibilities of effective treatment options for those living with PWS. 

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