Long-Term Safety and Effectiveness of Pitolisant Use in Adult Patients with Idiopathic Hypersomnia (IH)
Recommended Citation
Dauvilliers Y, Plante D, Corser B, Drake C, Bogan R, Insana S, Seiden D, Nomikos G, Wilmsen K, Manuel M, Budur K, Dayno J. Long-Term Safety and Effectiveness of Pitolisant Use in Adult Patients with Idiopathic Hypersomnia (IH). Sleep 2025; 48:A376-A377.
Document Type
Conference Proceeding
Publication Date
5-19-2025
Publication Title
Sleep
Abstract
Introduction: Pitolisant is being evaluated for use in adult patients with IH in two phase 3 clinical trials, a double-blind randomized withdrawal (DBRW) and an open-label extension (OLE). The present analyses focused on the safety and effective - ness of long-term administration of pitolisant in adult patients with IH across 13 months. The OLE study is ongoing. Methods: Open-label data were integrated across two con - tiguous phase 3 clinical trials (DBRW: NCT05156047; OLE: NCT05458128). Safety measures included treatment-related treatment-emergent adverse events (TR-TEAEs). Effectiveness measures included the Epworth Sleepiness Scale (ESS); Idiopathic Hypersomnia Severity Scale (IHSS); Patient Reported Outcomes Measurement Information System, Sleep Related Impairment-8a (PROMIS-SRI); Patient Sleep Inertia Questionnaire (SIQ); Functional Outcomes of Sleep Questionnaire (FOSQ-10); Patient Global Impression of Severity of EDS (PGI-S); and Clinician Global Impression of Severity of IH (CGI-S). Effectiveness measures were assessed at baseline, Month 2, Month 4, Month 10, and Month 16. Results: The safety population included 213 patients who received at least one dose of pitolisant (mean±SD age, 39.7±12.85 years; 79.3% female). The median duration of pitolisant exposure was 39.14 weeks (range: 0.71-93.29 weeks) and the median maximum pitolisant dosage was 35.6 mg (median [range: 8.9-35.6 mg]). Among this population, 102 participants (47.9%) experienced a TR-TEAE; most frequent TR-TEAEs were headache (17.8%), insomnia (16.0%), and abnormal dreams (5.2%). The effective - ness population included 119 adult patients who enrolled in both trials (mean±SD age, 40.1±11.96 years; 79.8% female). Generally, the sample size remained stable across timepoints. All effectiveness measures demonstrated a nominally statistically sig - nificant reduction (P< 0.0001) from study baseline at every time - point. Effectiveness measures are reported as baseline mean±SD, and change from baseline across all timepoints (range: lowest, highest change across endpoints [mean±SD]): ESS, 16.6±2.99 (-8.4±4.46, -9.7±4.69); IHSS, 33.7±7.10 (-9.8±7.56, -13.4±8.66); PROMIS-SRI, 64.16±4.89 (-8.35±7.26, -10.11±5.56); SIQ, 71.8±17.14 (-21.2±17.21, -24.9±18.91); FOSQ-10, 11.14±2.77 (3.31±2.99, 4.24±3.07); PGI-S, 3.9±0.74 (-1.2±0.99, -1.4±0.95); and CGI-S, 3.6±0.60 (-1.2±0.89, -1.3±0.78). Conclusion: Pitolisant was well-tolerated, and the safety profile was consistent with the established safety profile for patients with narcolepsy. Pitolisant showed clinically meaningful and sustained improvements in multiple symptoms of IH across a >1-year dosing period
Volume
48
First Page
A376
Last Page
A377
