Harmony Biosciences begins Phase 3 registrational study of pitolisant in PWS

Harmony Biosciences 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 FDA and their recent decision to grant Orphan Drug Designation for pitolisant in PWS. There is currently no FDA-approved treatment for EDS in this patient population. Pitolisant 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. PWS is an orphan/rare, genetic neurological disorder with many of the symptoms resulting from hypothalamic dysfunction.