FDA Broadens Pitolisant Label
The US Food and Drug Administration approves pitolisant (WAKIX) tablets for the treatment of cataplexy in pediatric patients aged 6 years and older with narcolepsy.
By
Kathryn Wighton
February 17, 2026
Clinical Scorecard: FDA Broadens Pitolisant Label
At a Glance
Category Detail
Condition Narcolepsy with or without cataplexy Key Mechanisms Selective histamine 3 receptor antagonist and inverse agonist, believed to increase histamine synthesis and release. Target Population Pediatric and adult patients aged 6 years and older Care Setting Outpatient clinical settings
Key Highlights
Only non-scheduled therapy approved for narcolepsy in the US. Indicated for excessive daytime sleepiness and cataplexy. Approved for pediatric patients aged 6 years and older. Received orphan drug designation in 2010 and breakthrough therapy designation in 2018. Narcolepsy affects approximately 170,000 people in the US. Guideline-Based Recommendations
Diagnosis
Diagnosis of narcolepsy based on clinical criteria including excessive daytime sleepiness and cataplexy. Management
Pitolisant can be prescribed for managing excessive daytime sleepiness and cataplexy. Monitoring & Follow-up
Monitor for QT prolongation and adverse reactions such as insomnia, nausea, and anxiety. Risks
Contraindicated in patients with hypersensitivity to pitolisant or severe hepatic impairment. Patient & Prescribing Data
Patients with narcolepsy aged 6 years and older.
Efficacy believed to be mediated through histamine 3 receptor activity.
Clinical Best Practices
Assess for history of cardiac arrhythmias before prescribing. Educate patients about potential side effects including insomnia and anxiety. Consider alternative therapies for patients with known QT prolongation. References
