Saturday, July 13, 2024

Narcolepsy Treatment Advances: FDA Approves Harmony Biosciences’ Wakix for Excessive Daytime Sleepiness in Pediatric Patients

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Narcolepsy treatment has advanced with the Food and Drug Administration (FDA) approving Harmony Biosciences’ Wakix (pitolisant) tablets for treating excessive daytime sleepiness (EDS) in pediatric patients aged 6 years and older. This decision marks a significant advancement in the management of narcolepsy, offering a new, non-scheduled treatment option for children. The approval is based on a Phase III study conducted by Bioprojet, which demonstrated the safety and efficacy of Wakix in patients over the age of 6.

The Phase III trial, a double-blind, multicenter, randomized, placebo-controlled study, included 110 participants from Finland, France, Italy, the Netherlands, and Russia. The study evaluated Wakix’s safety and efficacy in children aged 6 to less than 18 years with narcolepsy, with or without cataplexy. Following the initial trial phase, a prolonged open-label period allowed for further observation of the treatment’s effects.

Promising Narcolepsy Treatment Approved: Wakix Gains FDA Approval for Pediatric Use Despite Common Adverse Events

Common adverse events reported during the trial included insomnia, nausea, and anxiety. Other adverse events were headache, upper respiratory tract infection, musculoskeletal pain, increased heart rate, hallucinations, irritability, abdominal pain, sleep disturbance, decreased appetite, cataplexy, dry mouth, and rash. Despite these side effects, the trial’s results were promising, leading to the FDA’s approval of Wakix for pediatric use.

Wakix is notable for being the first non-scheduled treatment approved for EDS in pediatric patients. This approval provides a critical new option for children suffering from narcolepsy, who previously had limited treatment choices, all of which were controlled substances. Jeffrey M. Dayno, MD, President and CEO of Harmony Biosciences, expressed his satisfaction with the FDA’s timely review and approval, highlighting the importance of EDS as a primary symptom experienced by all narcolepsy patients.

“The approval of 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,” said Dayno.


Narcolepsy Treatment Advances: Wakix Targets Histamine System to Address Pediatric EDS Needs

Wakix works by targeting the histamine system to promote wakefulness, addressing a significant unmet need in pediatric EDS patients. Since its initial approval in the United States in 2019 for adults with EDS, and its subsequent approval in 2020 for adults with cataplexy, Wakix has shown a unique mechanism of action that is especially beneficial for pediatric populations.

“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. Harmony Biosciences is also working on next-generation formulations of Wakix, aiming to offer additional benefits such as greater efficacy and new indications, which could further extend the Wakix franchise and strengthen their leadership in treating rare sleep disorders.

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

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The approval of Wakix has been well-received by the narcolepsy community. Monica Gow, co-founder and executive director of Wake Up Narcolepsy, expressed her support for the FDA’s decision. “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 Gow. “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.”

The FDA’s approval of Wakix for treating EDS in pediatric patients with narcolepsy is a landmark achievement. It not only provides a new, effective treatment option for children suffering from this debilitating condition but also underscores the importance of innovative, non-scheduled medications in expanding therapeutic options for pediatric populations. Harmony Biosciences’ continued efforts to develop and improve treatments for rare sleep disorders promise further advancements in the future, offering hope and improved quality of life for patients and their families.


Resource: PR Newswire, June 24, 2024

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