Abstract Details

Title Safety and Tolerability of Pitolisant in the Treatment of Adult Patients With Narcolepsy: An Open-Label, Expanded Access Program in the United States

Topic Sleep

Presentation(s) P3 - Poster Session 3 (12:00 PM-1:00 PM)

Poster/Presentation
Number 5-002

Objective To examine the safety and tolerability of pitolisant in adults with narcolepsy.

Background 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.

Design/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) and the use of concomitant narcolepsy medications. Demographic and baseline information for all enrolled patients, and safety results available through August 28, 2019, are reported here (presentation will include updated data, if available).

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). Most patients (70.0%) were on treatment with ≥1 concomitant narcolepsy medication, including traditional stimulants (50.1%), sodium oxybate (31.0%), modafinil (14.3%), armodafinil (14.8%), and antidepressants (5.1%). Overall, 35.2% of patients have discontinued from the program; 16.7% due to an AE and 12.2% for lack of effect. A total of 573 AEs were reported, most commonly headache (10.8% of patients), nausea (7.2%), anxiety (5.9%), and insomnia (5.4%). AEs were generally mild or moderate in intensity (94.8% of AEs).

Conclusions In the PEACE program, patient characteristics were generally reflective of the US narcolepsy patient population. AEs were generally mild or moderate, and the tolerability profile 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.

Authors/Disclosures
Michael J. Thorpy, MD (Montefiore Medical Center) PRESENTER	Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Jazz. Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avadel. Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for alkermes. Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Harmony. Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving as a Consultant for centessa. Dr. Thorpy has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Axsome. Dr. Thorpy has received publishing royalties from a publication relating to health care.
	No disclosure on file
Craig Davis, PhD (Harmony Biosciences)	Dr. Davis has received personal compensation for serving as an employee of Harmony Biosciences. Dr. Davis has received stock or an ownership interest from Harmony Biosciences.
Albena I. Patroneva, MD (Harmony Biosciencesw)	Dr. Patroneva has nothing to disclose.
Jeffrey M. Dayno, MD (Harmony Biosciences, LLC)	Dr. Dayno has received personal compensation for serving as an employee of Harmony Biosciences. Dr. Dayno has received stock or an ownership interest from Harmony Biosciences.

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