Also known as: Wakix, WAKIX
Pitolisant is a histamine-3 receptor antagonist/inverse agonist marketed as Wakix for narcolepsy. It is used to treat excessive daytime sleepiness and cataplexy in patients aged 6 years and older. The FDA first approved Wakix on August 14, 2019.
The FDA has approved WAKIX for cataplexy in pediatric narcolepsy patients, making it the first non-scheduled treatment for all ages. Harmony Biosciences reported 2025 WAKIX revenue of $868.5 million and projects 2026 sales over $1 billion, nearing blockbuster status.
The FDA approved pitolisant (Wakix) for treating cataplexy in children ages 6 and older with narcolepsy, making it the first non-scheduled treatment for both pediatric and adult narcolepsy patients with or without cataplexy.
The FDA has expanded approval of Wakix (pitolisant) to treat cataplexy in pediatric patients aged 6 years and older with narcolepsy, making it the first non-controlled substance approved for both pediatric and adult narcolepsy patients with or without cataplexy.
The FDA has approved Harmony Biosciences' Wakix (pitolisant) for treating cataplexy in pediatric patients aged 6 years and older with narcolepsy, making it the first non-scheduled treatment for both pediatric and adult narcolepsy patients with or without cataplexy.
The FDA has approved pitolisant tablets for treating cataplexy in pediatric patients aged 6 and older with narcolepsy, making it the only non-scheduled therapy approved for both pediatric and adult narcolepsy patients with or without cataplexy.
The FDA approved Harmony Biosciences' supplemental new drug application for WAKIX (pitolisant) to treat cataplexy in pediatric patients 6 years and older with narcolepsy, making it the first non-scheduled treatment for both pediatric and adult narcolepsy patients with or without cataplexy.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07219485 |
A Study of Pitolisant in Participants With Prader-Willi Syndrome |
ENROLLING_BY_INVITATION | PHASE3 |
| NCT05953389 |
Proof of Concept Study on Pitolisant Effect on Autism Spectrum Disorders in Children and Adolescents |
ACTIVE_NOT_RECRUITING | PHASE2 |
| NCT05581576 |
Pitolisant in Refractory Restless Legs Syndrome |
UNKNOWN | PHASE4 |
| NCT05536011 |
WAKIX® (Pitolisant) Pregnancy Registry |
RECRUITING | |
| NCT05458128 |
A Long-Term Safety and Effectiveness Study to Evaluate Pitolisant in Adult Patients With Idiopathic Hypersomnia |
COMPLETED | PHASE3 |
| NCT05223166 |
A Clinical Study to Evaluate the Efficacy and Safety of Pitolisant in the Treatment of EDS in Patients With OSA |
COMPLETED | PHASE3 |
| NCT04596267 |
Pitolisant Effects on Alcohol Self-Administration in Heavy Drinkers |
TERMINATED | PHASE1 |
| NCT04026750 |
Insulin Tolerance Test Study in Patients With Type 1 Diabetes |
TERMINATED | PHASE1 |
| NCT03433131 |
Expanded Access Program to Provide Treatment With Pitolisant to Adult Patients in the U.S. With Excessive Daytime Sleepiness Associated With Narcolepsy With or Without Cataplexy |
NO_LONGER_AVAILABLE | |
| NCT03152123 |
Determination the Abuse Potential of Pitolisant in Healthy, Non-Dependent Recreational Stimulant Users |
COMPLETED | PHASE1 |
