The efficacy of Pitolisant-wakix

Pitolisant-wakix was investigated in two major studies involving a total of 261 adults with narcolepsy, most of whom also had cataplexy. These studies compared tilorisen tablets with placebo. The main measure of effectiveness is based on a patient's daytime sleepiness, assessed using the Epworth Sleepiness Scale, or ess, a standard scale used for people with narcolepsy that ranges from 0 to 24.

The first study showed that tilolixen tablets were more effective than placebo in reducing daytime sleepiness. After 8 weeks of treatment, patients taking tilolixen tablets experienced an average reduction of 3 points in ESS scores compared with patients taking placebo. Results from this study also showed a reduction in the number of cataplexy episodes. However, the second study did not show a difference between tilolixen tablets and placebo in reducing drowsiness or cataplexy. When drowsiness was measured using an objective test called the Stay Awake Test (MWT), the results from both studies together showed that Tilolixen significantly improved wakefulness compared to placebo. In a third study involving 105 patients with narcolepsy and cataplexy, tilolixen was also more effective than placebo in reducing the number of cataplexy episodes, with patients taking tilolixen reducing their number of cataplexy episodes from about nine to three per week.

The fourth study involved110 children aged 6 to 17 years old with narcolepsy, most of whom also had cataplexy. In this study, the Ullanlinna Narcolepsy Scale (UNS), a questionnaire with scores ranging from 0 to 44, was used to measure the frequency and severity of sleepiness and cataplexy. After eight weeks of treatment, children taking tilolixen tablets had an average UNS score that was 4 points lower than those taking placebo. In addition, patients taking tilolixen tablets had an average 2-point lower cataplexy score than those taking placebo.

Tilorisen tablets have been marketed in the country, but have not yet been included in medical insurance. The price of each box of the original version of tilorisen tablets marketed overseas may be over more than RMB 4,000 (the price may fluctuate due to exchange rates). Currently, there are no generic versions of tilorisen tablets produced and marketed overseas.