The TEMPO study will evaluate the safety and efficacy of pitolisant for treating excessive daytime sleepiness and behavioral symptoms in patients with Prader-Willi syndrome.

Harmony Biosciences Holdings Inc 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 6 years and older with Prader-Willi syndrome (PWS).

The TEMPO study initiation follows a successful end-of-phase 2 meeting with the US Food and Drug Administration (FDA) and their recent decision to grant Orphan Drug designation for pitolisant in PWS. Orphan Drug designation 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.

“The initiation of our phase 3 TEMPO study, a global, randomized, double-blind, placebo-controlled, multicenter trial with an open-label extension period, reflects continued positive momentum across our organization spanning our lifecycle management programs in PWS and other indications with high unmet medical need,” says Kumar Budur, MD, MS, chief medical officer of Harmony Biosciences, in a release. “With the initiation of this study and the FDA’s recent decision to grant [Orphan Drug designation] for pitolisant in PWS, we aim to potentially introduce a new, non-scheduled treatment option for EDS and the common behavioral symptoms in patients living with this condition.”

Approximately 15,000 to 20,000 people in the US are living with PWS, the majority experiencing behavioral symptoms and more than half with EDS, according to a release from Harmony Biosciences. There is currently no FDA-approved treatment for EDS in this patient population.

Pitolisant is marketed as Wakix in the US 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.