Pitolisant Receives FDA Approval for Pediatric Patients With Narcolepsy

The US Food and Drug Administration (FDA) recently approved Wakix (pitolisant) for the treatment of excessive daytime sleepiness in pediatric patients 6 years and older with narcolepsy, US maker Harmony Biosciences announced.

The supplemental New Drug Application for pitolisant received priority review based on a phase 3 study, by the French firm Bioprojet, that evaluated the safety and efficacy of pitolisant tablets in patients aged 6 to 17 years with narcolepsy with or without cataplexy. Bioprojet developed the drug and granted an exclusive license to Harmony Biosciences to manufacture and commercialize the drug in the United States.

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The FDA separated the supplemental New Drug Application for pitolisant for administrative purposes, according to Harmony Biosciences, to approve the drug for excessive daytime sleepiness in pediatric patients and to issue a response regarding the treatment of cataplexy in the patient population.  

“We plan to discuss with the agency a path forward for a cataplexy indication in pediatric narcolepsy patients based on the strength of the existing data from Bioprojet’s phase 3 trial,” said Kumar Budur, MD, MS, chief medical and scientific officer of Harmony Biosciences. “We appreciate the FDA’s recognition of the unmet medical need in this patient population and their approval of the excessive daytime sleepiness indication.”

Pitolisant is a first-in-class medication approved by the FDA in August 2019 for the treatment of excessive daytime sleepiness in adults with narcolepsy. In October 2020, the drug received an additional approval for the treatment of cataplexy in adults with narcolepsy. 

Pitolisant is a selective histamine 3 receptor antagonist/inverse agonist with an unclear mechanism of action. It is believed to target the histamine system to promote wakefulness.

“The unique mechanism of action of Wakix and its nonscheduled status are especially important for a pediatric population that has had limited treatment options, all of which are controlled substances,” said Jeffrey M. Dayno, MD, president and chief executive officer of Harmony Biosciences. “The unique features of pitolisant present an exciting opportunity, and we are currently working on the next-generation formulations that could potentially offer additional benefits to patients.”

Reference

Harmony Biosciences receives US Food And Drug Administration approval for Wakix® (pitolisant) in pediatric patients with narcolepsy. News release. Harmony Biosciences; June 24, 2024. Accessed July 19, 2024.