FDA approval for Wakix (pitolisant) in pediatric patients with narcolepsy

The FDA separated the submission into two 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 <18 years of age) with narcolepsy.

The FDA granted priority review of the sNDA based on a Phase III study conducted by Bioprojet, which evaluated the safety and efficacy of pitolisant in patients ages 6 to under 18 years with narcolepsy, with or without cataplexy. Based on the results of this study, Bioprojet received approval from the European Medicines Agency last year extending the indication for Wakix to include the treatment of narcolepsy in children ages 6 years of age and older, with or without cataplexy.

"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, M.D., 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 non-scheduled treatment option for narcolepsy, makes this important treatment option available to pediatric patients 6 years and older living with narcolepsy."

Wakix was first approved by the FDA in August 2019 for the treatment of EDS in adult patients with narcolepsy, followed by FDA approval for the treatment of cataplexy in adult patients with narcolepsy in October 2020. A first-in-class treatment with a novel mechanism of action, Wakix functions as a selective histamine 3 (H3) receptor antagonist/inverse agonist that is believed to target the histamine system to promote wakefulness.

Dayno added, "The unique mechanism of action of Wakix and its non-scheduled status are especially important for a pediatric population that has had limited treatment options, all of which are controlled substances. 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."

"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 III trial," said Kumar Budur, M.D., M.S., 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."