The European Medicines Agency (EMA) has approved Bioprojet’s Wakix (pitolisant) for the treatment of narcolepsy in children over 6 years of age, with or without cataplexy.
The expanded indication was based on a study of 110 patients, 72 of whom 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 showed that Wakix significantly reduces sleepiness and cataplexy after eight weeks of treatment compared to placebo, according to a press release by Bioprojet.
“The arrival of Wakix offers a new therapeutic option, with a favorable 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,” says Yves Dauvilliers, MD, PhD, 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, in a press release. “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 treatment of narcolepsy is based on:
- Behavioral 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, according to a release by Bioprojet.
EMA designated Wakix an orphan drug in 2007 for the treatment of narcolepsy. It is subject to a restricted medical prescription, reserved for sleep specialists.