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Harmony Biosciences to trial narcolepsy drug in Prader-Willi syndrome



A new clinical trial will evaluate whether the drug candidate, pitolisant, can help manage excessive daytime sleepiness in patients with Prader-Willi syndrome (PWS).

Harmony Biosciences has initiated its global Phase 3 trial to evaluate the safety and efficacy of pitolisant as a treatment for excessive daytime sleepiness (EDS) and behavioural symptoms in patients aged six years and older with the rare condition.

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 (ODD) for pitolisant in PWS.

Pitolisant is marketed as WAKIX® in the US and is FDA approved to treat EDS or cataplexy in adult patients with narcolepsy.

ODD incentivises 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 US are living with PWS, the majority experiencing behavioural symptoms and more than half with EDS.

PWS is a rare genetic neurological disorder with many of the symptoms resulting from hypothalamic dysfunction.

The hypothalamus is the part of the brain that controls both sleep-wake state stability and signals that mediate the balance between hunger and satiety, resulting in the main symptoms in patients with PWS, hyperphagia (an intense persistent sensation of hunger accompanied by food preoccupations, an extreme drive to consume food, food-related behaviour problems, and a lack of normal satiety), EDS and behavioural symptoms. Other features include low muscle tone, short stature, and cognitive impairment.

There is currently no FDA-approved treatment for EDS in this patient population.

Dr Kumar Budur, chief medical officer of Harmony Biosciences, said: “The initiation of our Phase 3 TEMPO study, a global, randomised, double-blind, placebo-controlled, multi-centre trial with an open-label extension period, reflects continued positive momentum across our organisation spanning our lifecycle management programs in PWS and other indications with high unmet medical need.

“With the initiation of this study and the FDA’s recent decision to grant ODD for pitolisant in PWS, we aim to potentially introduce a new, non-scheduled treatment option for EDS and the common behavioural symptoms in patients living with this condition.”

Dr Budur added: “Our progress in expanding the clinical applications of pitolisant in PWS and in other rare diseases, remains promising. These efforts are integral to our life cycle management programs, which, if successful, could benefit over 100,000 patients living with unmet medical needs.”