WAKIX® (Pitolisant) Receives Approval for the Treatment of Narcolepsy in Children Over 6 Years of Age, a Rare, Under-Diagnosed Condition

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 in the brain’s inability to regulate the normal sleep-wake cycle. It manifests as excessive daytime sleepiness, the irrepressible need to sleep, as well as disturbed night-time sleep, with rapid access, after falling asleep, to REM sleep. In childhood, it is associated with cataplexy attacks (sudden loss of muscle tone) in two thirds of patients.

The treatment of narcolepsy is based on:

• behavioural management, with recommendations for lifestyle (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 brain histamine H3 receptor for use in narcolepsy. Among the treatments for narcolepsy indicated for children, Wakix is the only non-narcotic, non-psychostimulant, non-addictive arousing 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 study showed a significant decrease in the “excessive daytime sleepiness-cataplexy” symptom score of 6.29 points versus -2.60 in the placebo group. 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.

According to the opinion of the European Committee for the Evaluation of Medicinal Products for Human Use (CHMP) dated 26 January 2023, “pitolisant significantly reduced daytime sleepiness compared to placebo, confirming its arousing 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.”