On 31 March 2016, the European Commission issued a decision for a marketing 
authorisation valid throughout the European Union (EU) for pitolisant (Wakix) 
for the treatment of narcolepsy with or without cataplexy in adults. Pitolisant 
is an antagonist/inverse agonist of the human histamine H3 receptor. The dose 
should be selected using an up-titration scheme depending on individual patient 
response and tolerance and should not exceed 36 mg/day. The main evidence of 
efficacy of pitolisant was based on two Phase III clinical trials. The 
improvement on excessive daytime sleepiness was shown against placebo in the 
Harmony I study (-3.33 points; 95% confidence interval (CI) [-5.83; -0.83]; 
p = 0.024) and in Harmony CTP (-3.41 points; 95% CI [-4.95; -1.87]; p < 0.0001). 
The daily cataplexy rate in Harmony I improved against placebo with a rate ratio 
(rR) of 0.38 whilst in the Harmony CTP the ratio of improvement on weekly 
cataplexy rate against placebo was 0.512. The most commonly reported adverse 
reactions were headache, insomnia and nausea. This article summarizes the 
scientific review leading to approval of pitolisant in the EU. The assessment 
report and product information are available on the European Medicines Agency 
website (http://www.ema.europa.eu).