Approved sNDA: Wakix for Treatment of Excessive Daytime Sleepiness in Youth With Narcolepsy

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Harmony Biosciences announced that the US Food and Drug Administration (FDA) has approved its supplemental New Drug Application (sNDA) for pitolisant (Wakix) tablets for the treatment of excessive daytime sleepiness (EDS) in pediatric patients aged 6 years and older with narcolepsy. The FDA then separated the submission into 2 sNDAs for administrative purposes to issue an approval for the treatment of EDS and a complete response for the treatment of cataplexy in pediatric patients (6 to under 18 years of age) with narcolepsy.¹

“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, MD, president and chief executive officer of Harmony Biosciences. “EDS is the primary symptom experienced by all patients with narcolepsy and this approval for WAKIX, as the first-and-only FDA-approved nonscheduled treatment option for narcolepsy, makes this important treatment option available to pediatric patients 6 years and older living with narcolepsy.”

The FDA granted priority review of the sNDA based on a phase 3 study conducted by Bioprojet, which evaluated the safety and efficacy of pitolisant in patients aged 6 to under 18 years with narcolepsy, with or without cataplexy. Wakix was first approved by the FDA in August 2019 for the treatment of EDS in adult patients with narcolepsy. It then received FDA approval for the treatment of cataplexy in adult patients with narcolepsy in October 2020.²

“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 Dayno. “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, such as greater efficacy and new indications, extend the Wakix franchise, and strengthen our leadership position in the treatment of rare sleep disorders.”

Wakix is a first-in-class treatment that functions as a selective histamine 3 receptor antagonist/inverse agonist, believed to target the histamine system to promote wakefulness.

“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 EDS indication, making WAKIX available to every appropriate pediatric patient 6 years and older living with narcolepsy.”

For Monica Gow, the cofounder and executive director of Wake Up Narcolepsy, this approval is especially exciting: “As a parent of a son who was diagnosed with narcolepsy in childhood, who continues to work in advocacy for all people living with narcolepsy of all ages, I applaud the FDA for approving Wakix in pediatric patients 6 years of age and older. This new approval offers a promising nonscheduled treatment option for children with narcolepsy, marking an important step forward in addressing the unmet medical needs of these children.”

References

1. Harmony Biosciences receives U.S. Food and Drug Administration approval for Wakix® (pitolisant) in pediatric patients with narcolepsy. News release. June 24, 2024. https://www.biospace.com/article/releases/harmony-biosciences-receives-u-s-food-and-drug-administration-approval-for-wakix-pitolisant-in-pediatric-patients-with-narcolepsy/

2. FDA approves expanded use of Wakix for narcolepsy. American Academy of Sleep Medicine. October 17, 2020. Accessed June 24, 2024. https://aasm.org/fda-approves-expanded-use-of-wakix-for-narcolepsy/