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PARIS, March 15, 2023 /PRNewswire/ — The European Medicines Agency has just granted a marketing authorization indication extension for WAKIX® (pitolisant), henceforth indicated for the treatment of narcolepsy in children to from 6 years, with or without cataplexy.
Narcolepsy is a rare disease, which is caused by the brain’s inability to regulate the normal sleep-wake cycle[1]. It is manifested by excessive daytime sleepiness, by an irrepressible need to sleep, as well as by disturbed night sleep, with rapid access, after falling asleep, to REM sleep[2]. In childhood, it is associated with cataplexy attacks (sudden loss of muscle tone) in two thirds of patients[3].
The treatment of narcolepsy is based on:
Wakix is the first and only drug in the class of brain histamine H3 receptor antagonists/inverse agonists used in narcolepsy. Among narcolepsy treatments indicated for children, Wakix is the only non-narcotic, non-psychostimulant, wake-promoting medication that carries no risk of addiction. Its efficacy is significant and has been demonstrated both in excessive daytime sleepiness and in cataplexy attacks in children.
“The arrival of Wakix offers a new therapeutic option, with a favorable safety profile, in the treatment of narcolepsy in children from 6 years of age, a rare disease with a strong impact on social life”, underlines Prof. Yves Dauvilliers, coordinator of the National Reference Center for Narcolepsy and Hypersomnia, Department of Neurology, Gui de Chauliac Hospital, University of Montpellier, Montpellier Institute of Neurosciences, Inserm. Narcolepsy, especially in children, is vastly underdiagnosed and diagnosed too late, so it should always be taken into account when a child falls asleep in class. Confirmation of the diagnosis allows for good management, limiting disability and improving quality of life.
The evaluation of Wakix in the treatment of narcolepsy in children was carried out as part of a randomized study in 110 patients: 72 were included in the pitolisant group and 38 in the placebo group. The study showed a significant decrease in the “excessive daytime sleepiness-cataplexy” symptom score of 6.29 points versus -2.60 in the placebo group[4]. Analysis of the study’s secondary endpoints shows that WAKIX significantly reduces somnolence and cataplexy after 8 weeks of treatment compared to placebo[5].
According to the opinion of the European Committee for the Evaluation of Medicinal Products for Human Use (CHMP) dated January 26, 2023, “pitolisant significantly reduced daytime sleepiness compared to placebo, confirming its exciting effect against EDS (excessive daytime sleepiness ) on the ESS (Epworth Sleepiness Scale) and demonstrating its anti-cataplectic effect when administered according to an individual titration scheme based on the individual benefit/tolerance ratio.[6]”.
About narcolepsy:
Narcolepsy usually begins in childhood and persists into adolescence and adulthood. The prevalence of narcolepsy in children is estimated to be between 0.02% and 0.06% in Western countries, a projection based on studies conducted in adult populations. The first symptoms of narcolepsy appear before the age of 18 in more than half of patients and sometimes begin before the onset of puberty, often with similar but stronger symptoms than in adults[7].
It is associated with comorbidities (rapid weight gain, precocious puberty and ADHD, depression, anxiety). It also has important repercussions on social life, and especially for the child, reducing their school performance.
About Wakix
Pitolisant is a histamine H3 receptor antagonist/inverse agonist discovered by Jean-Charles Schwartz, his collaborators at Inserm and the Bioprojet company in Paris, with the help of two European chemists (Walter Schunack, from the University of Berlin, and Robin Ganellin of University College London). By blocking histamine autoreceptors, pitolisant increases the activity of brain histaminergic neurons, an important arousal system with projections throughout the brain. Pitolisant also increases the release of acetylcholine, norepinephrine, and dopamine in the brain. This drug was already indicated in Europe and the United States for the treatment of EDS and cataplexy in adult patients with narcolepsy.
Wakix was designated an orphan drug in 2007 EU/3/07/459, for the treatment of narcolepsy. It is subject to a restricted medical prescription, reserved for sleep specialists.
It should be prescribed at the lowest effective dose, with a gradual increase in dose, not to exceed 20 mg (Wakix 18 mg), for patients weighing less than 40 kg.[8]
About Bioproject
The Bioprojet laboratory was born in 1982 thanks to the initiative of two researchers, Jeanne-Marie Lecomte and Jean-Charles Schwartz, whose purpose was to bring academic research closer to industrial pharmaceutical development, a practice not very widespread at that time.
Bioprojet pursues one goal: to rely on original fundamental research work to conceive and develop medicines that, like pitolisant, would be the first of new therapeutic classes.
Bioprojet is established in seven European countries and its research center conceives and studies a thousand new molecules every year.
www.bioprojet.com
[1] CHMP Extension of Indication Variation Assessment Report – Wakix , EMA, January 26, 2023, p. 7 [2] https://institute-of-sleep-vigilance.org/wp-content/uploads/2020/02/INSV-Narcolepsy-3-Volets.pdf [3] CHMP Extension of Indication Variation Assessment Report – Wakix , EMA, January 26, 2023, p. 8[4] CHMP Extension of Indication Variation Assessment Report – Wakix , EMA, 26 January 2023, p. 33[5] CHMP Extension of Indication Variation Assessment Report – Wakix , EMA, 26 January 2023, p. 9[6]Ibid.[7] CHMP Extension of Indication Variation Assessment Report – Wakix , EMA, January 26, 2023, p. 7[8] CHMP Extension of Indication Variation Assessment Report – Wakix , EMA, 26 January 2023 p. 10 –
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