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FDA Approves Harmony Biosciences’ Wakix to Treat Excessive Daytime Sleepiness in Pediatric Patients with Narcolepsy


Approval of Wakix marks the first time a non-scheduled treatment option for excessive daytime sleepiness has been approved for patients ages 6 years and older.

Excessive daytime sleepiness -Tired girl in sleep mask. Image Credit: Adobe Stock Images/Julia

Image Credit: Adobe Stock Images/Julia

The FDA has approved Harmony Biosciences’ Wakix (pitolisant) tablets for the treatment of excessive daytime sleepiness (EDS) in pediatric patients aged 6 years and older with narcolepsy. The FDA based the approval on results from a Phase III study conducted by Bioprojet, which evaluated the safety and efficacy of the treatment in patients over 6 years of age. Additionally, Wakix is the first non-scheduled treatment to be approved for EDS in pediatric patients.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, CEO, Harmony Biosciences, in a press 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."

The double-blind, multicenter, randomized, placebo-controlled Bioprojet trial evaluated the safety and efficacy of Wakix in children aged from 6 to less than 18 years with narcolepsy with/without cataplexy, followed by a prolonged open-label period. The trial included 110 participants from Finland, France, Italy, the Netherlands, and Russia.2

The most common adverse events (AEs) included insomnia, nausea, and anxiety. Additional AEs included headache, upper respiratory tract infection, musculoskeletal pain, heart rate increased, hallucinations, irritability, abdominal pain, sleep disturbance, decreased appetite, cataplexy, dry mouth, and rash.

Wakix was first approved in the United States in 2019 for the treatment of adults with EDS, with a second indication approved in 2020 for adults with cataplexy. Harmony Biosciences stated that Wakix targets the histamine system to promote wakefulness, addressing a significant need in pediatric patients with EDS.1

"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,” added Dayno, in the press 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."

Harmony cautions that Wakix is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation. Further, anaphylaxis has been reported, and the treatment is also contraindicated in patients with severe hepatic impairment. Patients with known QT prolongation should avoid the use of Wakix, and it should not be used in combination with other drugs known to prolong the QT interval. Patients with a history of cardiac arrhythmias or any other condition that may increase the risk of sudden death, such as symptomatic bradycardia, hypokalemia, or hypomagnesemia, should also avoid Wakix.1

"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," said Monica Gow, co-founder, executive director, Wake Up Narcolepsy, in the press 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."


1. HARMONY BIOSCIENCES RECEIVES U.S. FOOD AND DRUG ADMINISTRATION APPROVAL FOR WAKIX® (PITOLISANT) IN PEDIATRIC PATIENTS WITH NARCOLEPSY. PR Newswire. June 24, 2024. Accessed June 24, 2024. https://www.prnewswire.com/news-releases/harmony-biosciences-receives-us-food-and-drug-administration-approval-for-wakix-pitolisant-in-pediatric-patients-with-narcolepsy-302179754.html 

2. Efficacy and Safety of Pitolisant in Pediatric Narcoleptic Patients With or Without Cataplexy, Double-blind Study Followed by a Prolonged Open-label Period. ClinicalTrials.gov. Accessed June 24, 2024. https://classic.clinicaltrials.gov/ct2/show/NCT02611687

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