Pitolisant Post Hoc Analysis Shows Rapid Onset of Response for Narcolepsy Symptoms

As published in CNS Drugs, post hoc analysis of clinical trial data shows a 2- to 3-week time to onset of response to pitolisant (Wakix; Harmony Biosciences, Plymouth Meeting, PA).

Onset of response, measured as statistically significant change in the mean Epworth Sleepiness Scale (ESS) score, began at week 2 for participants in HARMONY 1 and week 3 for participants in HARMONY CTP. A significantly greater mean change in weekly rate of cataplexy was observed at week 2 for HARMONY CTP and week 5 for HARMONY 1 with further improvement seen with pitolisant through the end of treatment. A higher proportion of those treated with pitolisant vs placebo had improvement in excessive daytime sleepiness (EDS) beginning at week 3 and week 2 for cataplexy. 

"We are pleased to share the results from this post-hoc analysis that sheds light on the time course of response to pitolisant based on data from 2 phase 3 trials," said Harmony's chief medical officer, Jeffrey Dayno, MD "Even though narcolepsy is a chronic disorder, often requiring life-long therapy, both patients and healthcare professionals usually want to know the time to response after initiating treatment, which can help to manage patient expectations. This analysis provides the data to support that response to pitolisant begins in the first few weeks of therapy for some patients while, for others, it may take up to 8 weeks to experience a clinical response. This is also important to know as an adequate treatment trial is often required when managing patients with a chronic disorder like narcolepsy." 

Pitolisant is approved by the Food and Drug Administration (FDA) for the treatment of excessive daytime sleepiness or cataplexy in adults with narcolepsy and has been commercially available in since late 2019.he post hoc analysis included 61 participants from HARMONY 1 (pitolisant, n=31; placebo, n=30) and 105 participants from HARMONY CTP (pitolisant, n=54; placebo, n=51).Those treated with pitolisant had their dose titrated to a maximum of 35.6 mg/day. The onset of response was defined as the first timepoint of statistical difference between pitolisant and placebo.