Abstract
Introduction
Pitolisant Expanded Access Clinical Evaluation (PEACE) provided adult patients with narcolepsy access to treatment with pitolisant while it was an investigational medication in the United States.
Methods
Pitolisant was titrated to 35.6 mg/day (or the highest tolerable dose) over a 3-week period. Dose adjustments were permitted at the discretion of the treating physician based on patient response. Treating physicians followed their standard of care and were required to report adverse events (AEs). Demographic and baseline information for all enrolled patients, and safety results available through October 30, 2019, are reported here (presentation will include final data from the PEACE program).
Results
In all, 623 patients (67.9% female; 84.6% white; mean age, 40.0 years; narcolepsy type 1, 51.5%) were treated with pitolisant in the PEACE program. Nearly all patients (98.4%) had been previously treated with other narcolepsy medications (88.1% with ≥2 narcolepsy medications). Overall, 35.2% of patients discontinued from the program; 16.7% due to an AE and 12.2% for lack of effect. At Month 1, 97.3% of patients remained in the study, 88.2% at Month 3, 76.5% at Month 6, 66.9% at Month 9, and 55.0% at Month 12. In all, 256 (41.1%) patients experienced ≥1 AE; majority (52.5%) of these AEs occurred early in treatment (by Week 3). The most commonly reported AEs were headache (9.8% of AEs), nausea (6.6%), anxiety (5.6%), and insomnia (4.7%).
Conclusion
In the PEACE program, patient characteristics were generally reflective of the US narcolepsy patient population. The safety and tolerability profile of pitolisant was similar to that seen in the clinical development program, with no new safety signals identified. The program ceased enrollment in August 2019 after the US approval of pitolisant for the treatment of excessive daytime sleepiness in adult patients with narcolepsy.
Support
Harmony Biosciences, LLC.
0611 The Safety and Tolerability of Pitolisant in the Treatment of Excessive Daytime Sleepiness and Cataplexy in Adult Patients With Narcolepsy: An Open-Label, Expanded Access Program in the United States
