Harmony Biosciences receives U.S. Food and Drug Administration Orphan Drug designation for Prader-Willi syndrome treatment

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to Harmony Biosciences’ pitolisant for the treatment of Prader-Willi syndrome (PWS).

“The decision to grant Orphan Drug designation to pitolisant indicates that it could be a promising treatment option for people living with Prader-Willi syndrome,” said Kumar Budur, M.D., M.S., Chief Medical Officer at Harmony Biosciences. “This 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.”

FDA Orphan Drug designation incentivizes the advancement of promising therapies for rare diseases by providing tax credits for clinical development, waivers for user fees, and seven years of market exclusivity following drug approval. Approximately 15,000 – 20,000 people in the U.S. are living with PWS, the majority experiencing behavioral symptoms and more than half with excessive daytime sleepiness (EDS).

In the upcoming Phase 3 registrational TEMPO study, Harmony will assess the safety and efficacy of pitolisant in treating EDS and behavioral disturbances in PWS. This global study, anticipated to begin in Q1 2024, will be a randomized, double-blind, placebo-controlled trial in patients six years and older with PWS.

Dr. Budur added: “We are excited about our upcoming Phase 3 TEMPO study and the progress we have made to broaden the clinical utility of pitolisant not just in PWS but other rare diseases as part of our life cycle management programs that, if successful, could potentially help over 100,000 patients. On behalf of Harmony, I would like to thank all the patients and family members for participating in our clinical trials, as well as our investigators and site personnel for their commitment to advancing science.”

PWS is an orphan/rare, genetic neurological disorder with many of the symptoms resulting from hypothalamic dysfunction. The hypothalamus is the part of the brain that controls both sleep-wake state stability and signals that mediate the balance between hunger and satiety, resulting in the main symptoms in patients with PWS, hyperphagia (an intense persistent sensation of hunger accompanied by food preoccupations, an extreme drive to consume food, food-related behavior problems, and a lack of normal satiety), EDS and behavioral symptoms. Other features include low muscle tone, short stature, and cognitive impairment.

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