Once-daily Narcolepsy Treatment Efficacious for Reducing Severe Symptoms

Data shows pitolisant effectively reduces daytime sleepiness and cataplexy, two major burdens experienced by patients with narcolepsy.

In order to evaluate the efficacy of pitolisant in adults with high burden of narcolepsy symptoms, data were pooled from two randomized, placebo-controlled, 7- or 8-week studies of pitolisant (titrated to a potential maximum dose of 35.6 mg/day) in adults with narcolepsy.

According to the study, patients were assessed in three distinct cohorts including the Epworth Sleepiness Scale (ESS) baseline score ≥16, Maintenance of Wakefulness Test (MWT) sleep latency ≤8 min, and ≥15 cataplexy attacks per week groups.

The ESS cohort comprised 118 patients (pitolisant, n=60; placebo, n=58). The MWT group included 105 patients (pitolisant, n=59; placebo, n=46) and 31 patients were categorized into the cataplexy group (pitolisant, n=20; placebo, n=11).

According to authors of the study, “On the ESS, least-squares mean change from baseline was significantly greater for pitolisant (-6.1) compared with placebo (-2.3; P<.001).”

Furthermore, the study results show that significantly more pitolisant-treated patients were classified as treatment responders: ESS score reduction ≥3, 69.0% in the pitolisant group vs 35.1% in the placebo group (P=.001); final ESS score ≤10, 36.2% vs 10.5%, respectively (P=.005).

In the MWT group, mean sleep latency increased from 3.5 minutes to 10.4 minutes with pitolisant and from 3.4 minutes to 6.8 minutes with placebo (P=.017), according to the study. Results state that least-squares mean change in the weekly rate of cataplexy was significantly greater for pitolisant (−14.5; baseline, 23.9; final, 9.4) compared with placebo (−0.1; baseline, 23.1; final, 23.0; P=.004).

The most common adverse event with pitolisant noted in the results of the study was headache.

“Pitolisant, at once-daily doses up to 35.6 mg, was efficacious for reducing excessive daytime sleepiness and cataplexy in patients with severe narcolepsy symptom burden,” concluded authors of the study.

Reference:
Davis C W, Kallweit U, Schwartz J C, et al. Efficacy of pitolisant in patients with high burden of narcolepsy symptoms: pooled analysis of short-term, placebo-controlled studies. Sleep Med. 2021;81:210-217. doi:10.1016/j.sleep.2021.02.037.