PLYMOUTH MEETING, PA — In a significant development for patients with Prader-Willi syndrome (PWS), Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) recently announced the initiation of a global Phase 3 registrational trial, dubbed the TEMPO study, to evaluate the safety and efficacy of pitolisant in treating excessive daytime sleepiness (EDS) and behavioral symptoms associated with PWS in patients aged six years and older.
This move comes on the heels of a successful End-of-Phase 2 meeting with the U.S. Food & Drug Administration (FDA) and the agency’s recent decision to grant Orphan Drug Designation (ODD) for pitolisant for use in PWS—a rare genetic disorder characterized by a constant sense of hunger, developmental delays, and in many cases, EDS. The ODD status is a critical milestone, offering incentives like tax credits and seven years of market exclusivity post-approval, aimed at encouraging the development of treatments for rare diseases.
PWS affects approximately 15,000 – 20,000 people in the U.S., with the majority experiencing behavioral symptoms and over half suffering from EDS. Despite the prevalence and severity of these symptoms, there are currently no FDA-approved treatments for EDS in this patient population, highlighting a significant unmet medical need.
Kumar Budur, M.D., M.S., Chief Medical Officer of Harmony Biosciences, expressed optimism about the trial’s potential impact. “The initiation of our Phase 3 TEMPO study reflects continued positive momentum across our organization and our commitment to addressing indications with high unmet medical need,” Budur stated. He further emphasized the company’s goal to introduce a new, non-scheduled treatment option for EDS and common behavioral symptoms in patients living with PWS.
The TEMPO study is designed as a global, randomized, double-blind, placebo-controlled, multicenter trial with an open-label extension period, setting the stage for rigorous evaluation of pitolisant’s effectiveness and safety in this new patient population.
Pitolisant, marketed under the brand name WAKIX® in the U.S., is already FDA-approved to treat EDS or cataplexy in adult patients with narcolepsy. Its mechanism of action involves increasing histamine levels in the brain, which plays a crucial role in regulating wakefulness. The drug’s potential extension to PWS patients represents a promising avenue for those affected by the syndrome, offering hope for improved quality of life through better management of sleep-related and behavioral symptoms.
The broader implications of Harmony Biosciences’ efforts extend beyond immediate patient benefits. Successfully expanding pitolisant’s indication to include PWS could set a precedent for the pharmaceutical industry, illustrating the viability of repurposing existing drugs to address the needs of rare disease populations. Such strategies can significantly reduce development times and costs, providing a faster path to bringing much-needed therapies to market.
Moreover, the company’s focus on life cycle management programs underlines the importance of continuous innovation in drug development and the potential to unlock new therapeutic applications for existing medications. As Budur highlighted, these efforts could benefit over 100,000 patients living with unmet medical needs, marking a substantial contribution to the field of rare disease treatment.
As the TEMPO study progresses, industry watchers and the medical community alike will be keenly observing its outcomes, hopeful for a breakthrough that could transform the treatment landscape for PWS and potentially other rare conditions with similar challenges.
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