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Open-label pitolisant
Pitolisant is a selective histamine H3 receptor antagonist that increases wakefulness by blocking inhibitory histamine signaling in the brain.
Pitolisant is a selective histamine H3 receptor antagonist that increases wakefulness by blocking inhibitory histamine signaling in the brain. Used for Narcolepsy type 1 and type 2, Idiopathic hypersomnia.
At a glance
| Generic name | Open-label pitolisant |
|---|---|
| Sponsor | Harmony Biosciences Management, Inc. |
| Drug class | Histamine H3 receptor antagonist |
| Target | H3 receptor (histamine H3 receptor) |
| Modality | Small molecule |
| Therapeutic area | Neurology |
| Phase | Phase 3 |
Mechanism of action
Pitolisant antagonizes histamine H3 receptors, which are autoreceptors and heteroreceptors that normally suppress histamine neuron activity and wakefulness. By blocking these receptors, pitolisant enhances histaminergic neurotransmission, promoting arousal and reducing excessive daytime sleepiness. This mechanism is distinct from stimulants and addresses the underlying neurobiological deficit in narcolepsy.
Approved indications
- Narcolepsy type 1 and type 2
- Idiopathic hypersomnia
Common side effects
- Insomnia
- Headache
- Anxiety
- Irritability
- Nausea
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)
- 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)
- A Phase 3 Study to Assess the Safety and Efficacy of Pitolisant in Adult Patients With Idiopathic Hypersomnia (PHASE3)
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:
- Open-label pitolisant CI brief — competitive landscape report
- Open-label pitolisant updates RSS · CI watch RSS
- Harmony Biosciences Management, Inc. portfolio CI