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Pitolisant tablet
Pitolisant is a selective histamine H3 receptor antagonist that increases histamine release in the brain to promote wakefulness.
Pitolisant is a selective histamine H3 receptor antagonist that increases histamine release in the brain to promote wakefulness. Used for Narcolepsy type 1 and type 2, Excessive daytime sleepiness associated with narcolepsy.
At a glance
| Generic name | Pitolisant tablet |
|---|---|
| Also known as | pitolisant |
| Sponsor | Harmony Biosciences Management, Inc. |
| Drug class | Histamine H3 receptor antagonist |
| Target | H3 receptor |
| Modality | Small molecule |
| Therapeutic area | Neurology |
| Phase | Phase 3 |
Mechanism of action
By blocking H3 autoreceptors on histaminergic neurons in the hypothalamus, pitolisant removes negative feedback inhibition and enhances histamine neurotransmission. This increased histaminergic signaling promotes arousal and wakefulness, making it effective for conditions characterized by excessive daytime sleepiness. The drug crosses the blood-brain barrier and acts centrally without significant peripheral effects.
Approved indications
- Narcolepsy type 1 and type 2
- Excessive daytime sleepiness associated with narcolepsy
Common side effects
- Insomnia
- Headache
- Anxiety
- Nausea
- Irritability
Key clinical trials
- A Study of Pitolisant in Patients With Prader-Willi Syndrome (PHASE3)
- A Study of Pitolisant in Participants With Prader-Willi Syndrome (PHASE3)
- A Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension (PHASE2)
- A Study of HBS-201 (Pitolisant Delayed-release) (PHASE1)
- A Long-Term Safety and Effectiveness Study to Evaluate Pitolisant in Adult Patients With Idiopathic Hypersomnia (PHASE3)
- Proof of Concept Study on Pitolisant Effect on Autism Spectrum Disorders in Children and Adolescents (PHASE2)
- Efficacy and Safety of Pitolisant in Pediatric Narcoleptic Patients With or Without Cataplexy, Double-blind Study Followed by a Prolonged Open-label Period (PHASE3)
- Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1 (PHASE2)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Pitolisant tablet CI brief — competitive landscape report
- Pitolisant tablet updates RSS · CI watch RSS
- Harmony Biosciences Management, Inc. portfolio CI