Treatment response, observed at week 8 in HARMONY-1 and week 7 in HARMONY-CTP, was defined for EDS based on Epworth Sleepiness Scale (ESS) score reduction (≥3-point decrease from baseline or final score ≤10) and for cataplexy with at least 50% reduction from baseline to stable dose period in the weekly rate of cataplexy (WRC). Pitolisant was titrated over a 3-week period to a maximum potential dose of 35.6 mg/day, after which the dose remained stable.
1 “The analyses included both a traditional approach to presenting efficacy data, as well as one that underscores the clinical relevance of the findings, using NNT and effect sizes to capture overall clinical benefit.” "The analyses that we are presenting at the SLEEP conference this year highlight the strength of the efficacy data for Wakix,” Jeffrey Dayno, MD, chief medical officer, Harmony, said in a statement. The results, presented at the 2021 SLEEP Virtual Annual Meeting, June 10-13, show a low number needed-to-treat (NNT), large effect sizes, and statistically significant and clinically meaningful reduction in the frequency of cataplexy attacks with treatment of pitolisant. Data from 2 post-hoc analyses from the HARMONY-1 (NCT01067222) and HARMONY-CTP (NCT01800045) studies further demonstrate the treatment benefit pitolisant (Wakix Harmony Biosciences) has on excessive daytime sleepiness (EDS) and cataplexy in patients with narolepsy.