Pitolisant Gets Orphan Drug Status for Idiopathic Hypersomnia

Pitolisant is a selective histamine-3 receptor antagonist/inverse agonist, granted Orphan Drug designation for the treatment of idiopathic hypersomnia.

The Food and Drug Administration (FDA) has granted Orphan Drug designation to pitolisant for the treatment of idiopathic hypersomnia. 

Idiopathic hypersomnia is a rare neurologic sleep disorder with symptoms that include excessive daytime sleepiness, extreme difficulty waking, and severe brain fog. Pitolisant is a selective histamine-3 receptor antagonist/inverse agonist that works by increasing the synthesis and release of histamine, a wake-promoting neurotransmitter in the brain.

The Company is currently evaluating the efficacy and safety of pitolisant in adults with idiopathic hypersomnia in the phase 3 INTUNE study (ClinicalTrials.gov Identifier: NCT05156047). Study data are expected in the fourth quarter of 2023.

“The FDA’s decision to grant Orphan Drug designation reinforces our belief in pitolisant as a promising therapy for adult patients with IH, with the unique added benefit of it being a nonscheduled, once-daily treatment option working through histamine to improve wakefulness,” said Kumar Budur, MD, Chief Medical Officer at Harmony Biosciences.

Pitolisant is currently marketed under the brand name Wakix for the treatment of excessive daytime sleepiness or cataplexy in adults with narcolepsy.

This article originally appeared on MPR


Harmony Biosciences announces US Food & Drug Administration Orphan Drug designation for pitolisant for treatment of idiopathic hypersomnia. News release. Harmony Biosciences. September 7, 2023. https://www.prnewswire.com/news-releases/harmony-biosciences-announces-us-food–drug-administration-orphan-drug-designation-for-pitolisant-for-treatment-of-idiopathic-hypersomnia-301920012.html