Harmony Biosciences’ Narcolepsy Drug for Children Gets FDA Priority Review

Harmony Biosciences

PLYMOUTH MEETING, PA — Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) has announced that the U.S. Food and Drug Administration (FDA) has granted priority review to its supplemental New Drug Application (sNDA) for WAKIX® (pitolisant). This novel drug is intended for the treatment of excessive daytime sleepiness (EDS) or cataplexy in pediatric narcolepsy patients aged six and older.

The FDA’s decision fast-tracks the regulatory timeline for the drug with a Prescription Drug User Fee Act (PDUFA) target action date set for June 21, 2024. The move underscores the urgency for new treatment options for children living with this debilitating neurological condition.

“We are committed to expanding the utility of pitolisant for patients with high unmet medical needs,” said Jeffrey M. Dayno, M.D., President and Chief Executive Officer at Harmony Biosciences. “We look forward to collaborating with the FDA to potentially introduce a new, non-scheduled treatment option for children with narcolepsy.”

The FDA’s decision to grant priority review was based on a Phase 3 multicenter, randomized, placebo-controlled study conducted by Bioprojet. The study evaluated the safety and efficacy of pitolisant in pediatric patients with narcolepsy, with or without cataplexy. As a result of this study, Bioprojet received approval from the European Medicines Agency last year to extend the indication for pitolisant to include the treatment of narcolepsy in children aged six and older.

Narcolepsy is a rare, chronic neurological disease that affects sleep-wake state stability. It impacts approximately 170,000 Americans and is primarily characterized by excessive daytime sleepiness (EDS) and cataplexy, along with other manifestations of REM sleep dysregulation. The disease affects men and women equally, with typical symptom onset in adolescence or young adulthood. However, it can take up to a decade to be properly diagnosed.

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WAKIX® is a first-in-class medication that has already been approved by the FDA for the treatment of EDS or cataplexy in adult narcolepsy patients. It has been commercially available in the U.S. since Q4 2019. The drug works as a selective histamine 3 (H₃) receptor antagonist/inverse agonist. While its exact mechanism of action is unclear, it’s believed that WAKIX’s efficacy could be mediated through its activity at H₃ receptors, thereby increasing the synthesis and release of histamine, a neurotransmitter that promotes wakefulness.

Harmony Biosciences, a leading company based in Montgomery County, Pennsylvania, specializes in developing and delivering innovative treatments for rare neurological diseases. With this latest noteworthy development, the company once again showcases its unwavering dedication to enhancing the quality of life for individuals affected by neurological conditions.

The FDA’s priority review designation for WAKIX® indicates a notable advancement in combating pediatric narcolepsy. As the regulatory process unfolds, industry watchers will closely monitor Harmony Biosciences’ advancements in bringing this innovative treatment to the market, showcasing promising developments and contributing to improved healthcare outcomes.

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