On Sep. 7, 2023, Harmony Biosciences announced that the FDA granted orphan drug designation to pitolisant for the treatment of idiopathic hypersomnia (IH). The FDA’s orphan drug designation provides incentives for drugs and biologics for the treatment of rare diseases.
About pitolisant
Pitolisant is a selective histamine 3 receptor antagonist/inverse agonist. It is marketed as WAKIX and is approved by the FDA for the treatment of excessive daytime sleepiness or cataplexy in adults with narcolepsy. Pitolisant is not yet approved for IH.
According to a press release, Harmony is evaluating the safety and efficacy of pitolisant in adults with IH in a double-blind, placebo-controlled, randomized withdrawal study. Topline study results are expected in fall 2023.
About idiopathic hypersomnia
IH is a rare sleep disorder characterized by excessive daytime sleepiness despite normal nighttime sleep duration and quality. Individuals with IH may experience difficulty waking up, exhibit confusion or a “sleep drunkenness” state upon awakening, and may find their prolonged daytime naps to be unrefreshing, posing potential risks in activities requiring alertness, such as driving or operating machinery.
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