PLYMOUTH MEETING, PA — The U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to Harmony Biosciences Holdings, Inc.’s (Nasdaq: HRMY) pitolisant for the treatment of Prader-Willi syndrome (PWS), a rare genetic neurological disorder. The announcement marks a significant step forward in Harmony’s ongoing efforts to address the high unmet medical needs within the PWS patient community.
Orphan Drug designation is awarded by the FDA to encourage the development of drugs for rare diseases. It provides incentives such as tax credits for clinical research, fee waivers, and seven years of market exclusivity following the drug’s approval. This recognition for pitolisant indicates its potential as a promising treatment option for PWS, which affects approximately 15,000 to 20,000 people in the U.S.
“The decision to grant Orphan Drug designation to pitolisant represents a promising advancement for people living with Prader-Willi syndrome,” said Kumar Budur, MD, MS, Chief Medical Officer at Harmony Biosciences. “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.”
PWS is characterized by symptoms resulting from hypothalamic dysfunction. The hypothalamus is a part of the brain that controls sleep-wake state stability and hunger-satiety balance. Patients with PWS often experience hyperphagia—an intense persistent sensation of hunger accompanied by preoccupation with food, an extreme drive to consume food, and a lack of normal satiety—along with excessive daytime sleepiness (EDS) and behavioral symptoms. Other features include low muscle tone, short stature, and cognitive impairment.
Harmony Biosciences plans to assess the safety and efficacy of pitolisant in treating EDS and behavioral disturbances in PWS patients in the upcoming Phase 3 TEMPO study. This randomized, double-blind, placebo-controlled trial will include patients six years and older with PWS and is expected to begin in Q1 2024.
“We are eager to continue working with the FDA and the broader community of PWS patients and caregivers,” Dr. Budur added. “Our life cycle management programs could potentially help over 100,000 patients if successful.”
Established by Paragon Biosciences, LLC, in 2017, Harmony Biosciences is headquartered in Montgomery County, Pennsylvania. The company specializes in developing and delivering treatments for rare neurological diseases that are often overlooked by others. Its team of experts is driven by the shared conviction that innovative science translates into therapeutic possibilities for patients, who are at the center of all their efforts.
In securing the FDA’s Orphan Drug designation for pitolisant, Harmony Biosciences aims t to address the unmet needs of the PWS community. As the company prepares for its Phase 3 TEMPO study, it remains poised to make significant strides in PWS treatment and potentially improve the lives of thousands of patients living with this rare disorder.
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