PLYMOUTH MEETING, PA — Harmony Biosciences (Nasdaq: HRMY) announced that the U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application (sNDA) for WAKIX® (pitolisant) tablets. The approval covers the treatment of excessive daytime sleepiness (EDS) in pediatric patients aged six and older with narcolepsy.
The FDA granted priority review of the sNDA following a Phase 3 study by Bioprojet. This study assessed the safety and efficacy of pitolisant in patients aged six to under 18 years with narcolepsy, with or without cataplexy. Based on the study, the European Medicines Agency had already approved WAKIX for children last year.
“Following the FDA’s decision to grant priority review, we are very pleased with the Agency’s timely review and approval of WAKIX for pediatric narcolepsy patients with excessive daytime sleepiness,” said Jeffrey M. Dayno, President and CEO of Harmony Biosciences.
WAKIX, first approved for adults in 2019, works as a selective histamine 3 (H₃) receptor antagonist/inverse agonist, promoting wakefulness without being a controlled substance. This feature is crucial for pediatric patients who previously had limited, controlled-substance treatment options.
Dayno highlighted the significance of WAKIX’s unique mechanism. “We are currently working on next-generation formulations that could potentially offer additional benefits to patients,” he said.
Chief Medical and Scientific Officer Kumar Budur emphasized plans to seek further FDA approval for cataplexy in pediatric patients, based on existing Phase 3 data. “We appreciate the FDA’s recognition of the unmet medical need in this patient population,” Budur stated.
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