Harmony Bio Starts Phase 3 in Prader-Willi Trials

  Harmony Biosciences Holdings, Inc. ("Harmony") (Nasdaq: HRMY) 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.

https://www.biospace.com/article/releases/harmony-biosciences-initiates-global-phase-3-registrational-trial-tempo-study-of-pitolisant-in-patients-with-prader-willi-syndrome/