The FDA’s approval extends the treatment, previously available for adults, to children aged 6 and older with excessive daytime sleepiness due to narcolepsy.

Summary: The FDA has expanded the approval of a narcolepsy drug, previously approved for adults, to now include children aged 6 and older. This new approval provides a non-scheduled treatment option for pediatric patients experiencing excessive daytime sleepiness due to narcolepsy.

Key Takeaways:

  • The FDA has extended the indication of a previously approved narcolepsy drug to include children aged 6 and older, offering a new treatment option for excessive daytime sleepiness in pediatric patients.
  • This approval introduces a non-scheduled treatment option for children with narcolepsy.
  • The expanded approval followed a priority review based on a phase 3 study that demonstrated the safety and efficacy of the drug in children.

The US Food and Drug Administration (FDA) has approved Harmony Biosciences’ Wakix (pitolisant) for treating excessive daytime sleepiness (EDS) in children aged 6 and older with narcolepsy.

The FDA separated Harmony’s supplemental new drug application (sNDA) 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 under 18 years of age) with narcolepsy.  The FDA will send a complete response letter if the agency determines that it will not approve the application or abbreviated application in its present form.

Harmony plans to discuss with the FDA a path forward for a cataplexy indication in pediatric narcolepsy patients based on existing data from Bioproject’s phase 3 trial. 

Treating EDS in Pediatric Patients 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 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,” says Jeffrey M. Dayno, MD, president and chief executive officer of Harmony Biosciences, in a release. “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 receptor antagonist/inverse agonist that is believed to target the histamine system to promote wakefulness.

Mechanism and Future Prospects

“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,” says Dayno in a release. “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.”

“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,” says Monica Gow, co-founder and executive director of Wake Up Narcolepsy, in a release. “This new approval offers a promising non-scheduled treatment option for children with narcolepsy, marking an important step forward in addressing the unmet medical needs of these children.”

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