Harmony Biosciences Launches TEMPO: Phase 3 Trial for PWS Patients

Harmony Biosciences has launched a pivotal Phase 3 clinical trial, known as TEMPO, to assess the impact of pitolisant on excessive daytime sleepiness (EDS) and behavioral issues in individuals aged six and above with Prader-Willi syndrome (PWS). The study follows a productive meeting with the FDA and the granting of Orphan Drug Designation (ODD) for pitolisant in PWS, which offers incentives such as tax credits and market exclusivity to encourage the development of treatments for rare conditions.
The TEMPO study is a global, randomized, double-blind, placebo-controlled trial with an open-label extension phase. It is designed to evaluate the safety and efficacy of pitolisant as a potential new treatment option for EDS and behavioral symptoms associated with PWS. PWS is a rare genetic disorder that affects the hypothalamus, leading to symptoms such as hyperphagia, EDS, and behavioral issues. Currently, there are no FDA-approved treatments for EDS in PWS patients, and Harmony Biosciences aims to fill this gap with pitolisant, which is already approved to treat EDS or cataplexy in adults with narcolepsy under the brand name WAKIX.
Dr. Kumar Budur, Harmony Biosciences' Chief Medical Officer, highlighted the significance of the study, stating that it represents a positive step forward in addressing the unmet medical needs of patients with PWS and other conditions. The company's efforts to expand the use of pitolisant in PWS and other rare diseases are part of a broader strategy to improve the lives of over 100,000 patients.
WAKIX, developed by Bioprojet in France and licensed to Harmony Biosciences for development and commercialization in the U.S., is a selective histamine 3 (H3) receptor antagonist/inverse agonist. Its mechanism of action is not fully understood, but it is believed to increase the synthesis and release of histamine, a neurotransmitter that promotes wakefulness.
The company emphasizes that WAKIX is not yet approved for use in PWS patients and is still under investigation. Harmony Biosciences is committed to developing treatments for neurological disorders that are often overlooked by others, with a focus on innovation and empathy to improve the quality of life for patients.