FDA Approves Harmony Biosciences' Wakix® for Pediatric Narcolepsy

Harmony Biosciences, headquartered in Plymouth Meeting, Pennsylvania, has announced that the U.S. Food and Drug Administration (FDA) has approved its supplemental New Drug Application (sNDA) for WAKIX® (pitolisant) tablets. This approval extends the use of WAKIX to treat excessive daytime sleepiness (EDS) in pediatric patients aged six and older who have narcolepsy. The FDA divided the sNDA submission into two parts to facilitate an approval for EDS treatment and a complete response for treating cataplexy in the same age group.

The FDA granted priority review for this sNDA based on a Phase 3 study conducted by Bioprojet, which evaluated the safety and efficacy of pitolisant in patients aged 6 to 18 with narcolepsy, with or without cataplexy. Last year, Bioprojet received approval from the European Medicines Agency to extend WAKIX's indication to include treating narcolepsy in children aged six and above.

Dr. Jeffrey M. Dayno, President and CEO of Harmony Biosciences, expressed satisfaction with the FDA's timely approval of WAKIX for pediatric narcolepsy patients experiencing excessive daytime sleepiness. He highlighted that EDS is a primary symptom for all narcolepsy patients and that WAKIX is the first FDA-approved non-scheduled treatment for this condition, now available to pediatric patients.

WAKIX was initially approved by the FDA in August 2019 for treating EDS in adults with narcolepsy, and later in October 2020 for treating cataplexy in the same patient group. The drug operates as a selective histamine 3 (H₃) receptor antagonist/inverse agonist, promoting wakefulness by targeting the histamine system.

Dr. Dayno emphasized the significance of WAKIX's unique mechanism of action and its non-scheduled status for a pediatric population with limited treatment options, all of which are typically controlled substances. He mentioned ongoing efforts to develop next-generation formulations that could enhance efficacy and offer new indications, thereby strengthening Harmony Biosciences' leadership in treating rare sleep disorders.

Dr. Kumar Budur, Chief Medical and Scientific Officer of Harmony Biosciences, stated that the company plans to discuss with the FDA a pathway for a cataplexy indication in pediatric narcolepsy patients, based on data from Bioprojet's Phase 3 trial. He acknowledged the FDA's recognition of the unmet medical need in this patient population and their approval of the EDS indication, making WAKIX accessible to appropriate pediatric patients.

Monica Gow, Co-Founder and Executive Director of Wake Up Narcolepsy, praised the FDA's decision, noting the importance of this new non-scheduled treatment option for children with narcolepsy. She highlighted that this approval marks a significant step in addressing the unmet medical needs of these children.

Narcolepsy is described as a rare, chronic neurological disorder characterized by sleep-wake state instability. It affects approximately 170,000 Americans and is marked by excessive daytime sleepiness, with or without cataplexy. Other symptoms include hallucinations and sleep paralysis, which are manifestations of REM sleep dysregulation. The condition is caused by the loss of hypocretin/orexin, a neuropeptide that stabilizes sleep-wake states. Symptom onset typically occurs during adolescence or young adulthood, and proper diagnosis can take up to a decade.

WAKIX, a first-in-class medication, is approved by the FDA for treating EDS or cataplexy in adults with narcolepsy and for treating EDS in pediatric patients aged six and older with narcolepsy. It was granted orphan drug designation for narcolepsy treatment in 2010 and breakthrough therapy designation for cataplexy treatment in 2018. Developed by Bioprojet in France, Harmony Biosciences holds an exclusive license to develop, manufacture, and commercialize pitolisant in the United States.