Treatment with pitolisant is associated with reductions in excessive daytime sleepiness (EDS) and cataplexy over a 1-year period in patients with narcolepsy, according to research presented at World Sleep 2019, held September 20 to 25, 2019, in Vancouver, Canada.
Patients with narcolepsy with or without cataplexy (mean age, 36 years) were enrolled in the pragmatic, open-label, multicenter HARMONY-3 trial (n=102). The trial is a long-term study that includes up to 5 years of follow-up data; however, only the 1-year results of the trial were presented at the meeting. In the 1-year data analysis, patients with EDS were given a maximum individualized once-daily pitolisant dose of 35.6 mg. A total of 73 patients in the study did not have a history of pitolisant use.
The mean Epworth Sleepiness Scale (ESS) at baseline was 17.1±3.1, and 73.5% of patients had cataplexy. A total of 68 patients completed ≥1 year of pitolisant therapy. Overall, the mean reduction in ESS was 4.3 points, including 4.9 (P <.01) points in patients without a history of pitolisant treatment and 4.2 points in previously treated patients.
The majority of patients (63.2%) were considered responders to therapy (ESS final ≤10 and/or ESS baseline – ESS final ≥3), and more than one-third of patients (36.8%) were normalized (ESS final ≤10). In normalized patients, the mean ESS was reduced from 15.3 to 6.6. Reductions in partial and total cataplexy attacks (-65% and -76%, respectively) as well as reductions in hypnagogic hallucinations (-54%) and sleep paralysis (-62%) at 1 year were also reported.
In the 56.9% of patients who reported adverse events during the 1-year pitolisant treatment period, the most common events included headaches (11.8%), insomnia (8.8%), weight gain (7.8%), anxiety (6.9%), depression (4.9%), and nausea (4.9%).
A limitation of the study included the small sample size. Despite these limitations, researchers concluded that “this 1-year analysis of a long-term, open-label study supports the safety and efficacy of pitolisant for the treatment of EDS and cataplexy in adult patients with narcolepsy.”
Disclosure: This study was funded and sponsored by Bioprojet Pharma.
Dauvilliers Y, Arnulf I, Scart-Grès C, et al. Long-term evaluation of safety and efficacy of pitolisant in narcolepsy: Harmony 3 study. Abstract presented at World Sleep 2019; September 20-25, 2019; Vancouver, Canada.