Wakix Shows Potential as Treatment for Individuals with Idiopathic Hypersomnia

A comprehensive analysis of 2 phase 3 clinical trials demonstrated that Wakix (pitolisant; Harmony Biosciences, Plymouth Meeting, PA) maintained a favorable safety profile and provided clinically meaningful improvements for multiple symptoms in adults diagnosed with idiopathic hypersomnia (IH). The integrated analysis of the INTUNE study (NCT05156047) and its open-label extension (OLE) (NCT05458128) revealed sustained effectiveness across patient-reported outcomes and clinician assessments with safety findings consistent with the established profile for narcolepsy patients. Study results were presented at the 2025 Annual Meeting of the Associated Professional Sleep Societies (APSS).

The safety analysis included 213 individuals diagnosed with IH (mean±SD age 39.7±12.85 years; 79.3% female) who received at least one dose of Wakix. The effectiveness analysis included 119 people (mean±SD age 40.1±11.96 years; 79.8% female) who enrolled in both trials, with assessments conducted at baseline and months 2, 4, 10, and 16. The median Wakix exposure was 39.14 weeks, and the median maximum dosage was 35.6 mg.

Statistically significant reductions in effectiveness measures were reported for all outcomes at all timepoints (reported as baseline mean ±SD and change from basline across all timepoints): 

• Epworth Sleepiness Scale (ESS): mean±SD score of 16.6±2.99 at baseline (-8.4±4.46 lowest change from baseline, -9.7±4.69 highest change from baseline)
• Idiopathic Hypersomnia Severity Scale (IHSS): 33.7±7.10 (-9.8±7.56, -13.4±8.66)
• Patient-Reported Outcomes Measurement Information System, Sleep Related Impairment-8a (PROMIS-SRI): 64.16±4.89 (-8.35±7.26, -10.11±5.56)
• Patient Sleep Inertia Questionnaire (SIQ): 71.8±17.14 (-21.2±17.21, -24.9±18.91)
• Functional Outcomes of Sleep Questionnaire (FOSQ-10): 11.14±2.77 (3.31±2.99, 4.24±3.07)
• Patient Global Impression of Severity of EDS (PGI-S): 3.9±0.74 (-1.2±0.99, -1.4±0.95)
• Clinician Global Impression of Severity of IH (CGI-S): 3.6±0.60 (-1.2±0.89, -1.3±0.78)

Treatment-related adverse events occurred in 47.9% of patients, most commonly headache (17.8%), insomnia (16.0%), and abnormal dreams (5.2%).