Bioprojet: WAKIX® (pitolisant) receives approval for the treatment of narcolepsy in children over 6 years of age, a rare, under-diagnosed condition
PARIS, March 15, 2023 /PRNewswire/ -- The European Medicines Agency has just granted an extension of the indication for the marketing authorisation for WAKIX® (pitolisant), which is now indicated for the treatment of narcolepsy in children from the age of 6 years, with or without cataplexy.
Narcolepsy is a rare disease, which originates from the brain's inability to regulate the normal sleep-wake cycle[1]. It manifests as excessive daytime sleepiness, the irrepressible need to sleep, cataplexy attacks (sudden loss of muscle tone), as well as disturbed night-time sleep, with rapid access, to REM sleep, after falling asleep[2]. In childhood, it is associated with more frequent cataplexy attacks in two thirds of patients[3].
The treatment of narcolepsy is based on:
- behavioural management, with recommendations for lifestyle management (regular bedtimes, scheduled naps, physical activity and appropriate diet);
- prescription of medication, especially for daytime sleepiness and cataplexy.
Wakix is the first and only drug in the class of antagonists/reverse agonists of the histamine H3 receptor from the cerebral nervous system for use in narcolepsy. Among the treatments for narcolepsy indicated for children, Wakix is the only non-narcotic, non-psychostimulant, almost non-addictive awakening drug. Its effectiveness is significant and proven both for excessive daytime sleepiness and cataplexy attacks in children.
"The arrival of Wakix offers a new therapeutic option, with a favourable safety profile, in the treatment of narcolepsy in children from the age of 6 years, a rare disease with a strong impact on social life," emphasises Prof. Yves Dauvilliers, coordinator of the National Reference Centre for Narcolepsy-Hypersomnia, Department of Neurology, Gui de Chauliac Hospital, University of Montpellier, Montpellier Institute of Neurosciences and Inserm. Narcolepsy, especially in children, is widely under-diagnosed, diagnosed too late, and should always be considered when a child is sleeping in class. Confirming the diagnosis allows good management, limiting disability and improving quality of life".
The evaluation of Wakix in the treatment of narcolepsy in children was conducted in a randomised study of 110 patients: 72 were included in the pitolisant group and 38 in the placebo group. The primary endpoint was the Ullanlinna Narcolepsy Score (UNS) assessing excessive daytime sleepiness and cataplexy attacks. The study showed a significant decrease in the UNS score - 6.29 points in the pitolisant group. versus -2.60 in the placebo group[4]. The analysis of the secondary endpoints of the study shows that WAKIX significantly reduces sleepiness 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 26 January 2023,in this study, with UNS and different scales, pitolisant significantly reduced daytime sleepiness and cataplexy attacks compared with placebo, confirming its wakefulness and anti-cataplectic effect when administered according to an individual titration scheme based on the individual benefit/tolerance ratio; . [6]
About narcolepsy:
Narcolepsy often begins in childhood and persists into adolescence and adulthood. The prevalence of narcolepsy in children is estimated at 0.02% to 0.06% in Western countries, a projection based on studies of adult populations. The first symptoms of narcolepsy occur before the age of 18 in more than half of patients and sometimes start before the onset of puberty, often with similar but stronger symptoms than in adults[7].
It is associated with co-morbidities (rapid weight gain, early puberty and ADHD, depression, anxiety). It also has a major impact on social life, with the child's performance at school often being impaired.
About Wakix
Pitolisant is a histamine H3 receptor antagonist/inverse agonist discovered by Jean-Charles Schwartz, his collaborators at Inserm and Société Bioprojet de Paris, with the assistance of two European chemists (Walter Schunack at Berlin University and Robin Ganellin at University College London). By blocking histamine autoreceptors, it increases the activity of cerebral histamine neurons, a major wakefulness system with projections extending to the entire brain. Pitolisant also increases the release of acetylcholine, noradrenaline and dopamine in the brain. Pitolisant is already indicated in Europe and the United States for the treatment of EDS and cataplexy in adult narcolepsy patients.
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 exceeding 20 mg (Wakix 18 mg), for patients weighing less than 40 kg.[8]
About Bioprojet
Bioprojet laboratory was formed in 1982 under the initiative of two researchers, Jeanne-Marie Lecomte and Jean-Charles Schwartz, in order to bring together academic research and commercial pharmaceutical development, a not very widespread practice at the time.
Bioprojet's objective: to rely on original fundamental research to design and develop medicines which, like pitolisant, would be the first of new therapeutic classes.
Bioprojet is based in seven European countries and its research centre designs and investigates a thousand new molecules every year.
[1] CHMP extension of indication variation assessment report - Wakix , EMA, 26 January 2023, p. 7
[2] https://institut-sommeil-vigilance.org/wp-content/uploads/2020/02/INSV-Narcolepsie-3-Volets.pdf
[3] CHMP extension of indication variation assessment report - Wakix , EMA, 26 January 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] CHMP extension of indication variation assessment report - Wakix , EMA, 26 January 2023, p. 69
[7] CHMP extension of indication variation assessment report - Wakix , EMA, 26 January 2023, p. 7
[8] CHMP extension of indication variation assessment report - Wakix , EMA, 26 January 2023 p. 10 - 14
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