US-based Harmony Biosciences has announced Phase III plans of its pipeline drug pitolisant, a potential treatment for excessive daytime sleepiness (EDS) in patients with Prader-Willi syndrome (PWS), which will include children, adolescents and adults.
Harmony now has plans to initiate the Phase III study in Q4 2023 following a successful end-of-Phase II meeting with the US Food and Drug Administration (FDA).
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The Phase III study design supports further investigation of pitolisant for children, adolescents and adults with PWS experiencing EDS. There is currently no FDA-approved treatment for EDS in this patient population.
Marketed for narcolepsy
Pitolisant is marketed as WAKIX in the US and has been commercially available since Q4 2019 to treat EDS or cataplexy in adult patients with narcolepsy.
However, pitolisant is not yet approved for use in patients with PWS. Harmony is also testing the drug for adult patients with idiopathic hypersomnia (IH).
Chief medical officer Dr Kumar Budur said: “We are pleased with the outcome of our end-of-Phase II meeting with the FDA as we prepare to initiate our Phase III registrational study, which aims to further investigate the efficacy and safety of pitolisant as a potential treatment for excessive daytime sleepiness in individuals with Prader-Willi syndrome. Building upon the encouraging data obtained from our Phase II signal detection study, we remain committed to advancing our development programme for pitolisant in pursuit of a new indication in people with PWS, given the high unmet medical need in this population.”
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By GlobalDataIn the US alone, 15,000 to 20,000 people live with PWS, a rare genetic neurological disorder often diagnosed in childhood. More than half of patients experience EDS, and many of the symptoms result from hypothalamic dysfunction. The hypothalamus controls both sleep-wake state stability and signals that mediate the balance between hunger and satiety. It results in two of the main symptoms in patients with PWS, EDS and hyperphagia.
About pitolisant
Pitolisant is a selective histamine 3 (H₃) receptor antagonist/inverse agonist. It was designed and developed by Bioprojet in France. Harmony has a licence from Bioprojet to develop, manufacture and commercialise pitolisant in the US.
Pitolisant is contraindicated in patients with known hypersensitivity to pitolisant, as well as any component of the formulation. Another contraindication is in patients with severe hepatic impairment. Anaphylaxis has been reported for the drug.
