Harmony Biosciences Holdings, Inc. has initiated its global Phase 3 registrational trial, the TEMPO study, to evaluate the safety and efficacy of pitolisant as a treatment for excessive daytime sleepiness (EDS) and behavioral symptoms in patients aged six years and older with Prader-Willi syndrome (PWS). The TEMPO study initiation follows a successful End-of-Phase 2 meeting with the U.S. Food & Drug Administration (FDA) and their recent decision to grant Orphan Drug Designation (ODD) for pitolisant in PWS. ODD 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 EDS. There is currently no FDA-approved treatment for EDS in this patient population.

Pitolisant is marketed as WAKIX® in the U.S. and is FDA approved to treat EDS or cataplexy in adult patients with narcolepsy. Pitolisant is not approved for use in patients with PWS and is currently being evaluated as an investigational agent in this patient population.