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Double-blind 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 | Double-blind 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
Pitolisant works by antagonizing H3 receptors on histaminergic neurons, which normally provide negative feedback to suppress histamine release. By blocking this inhibition, the drug enhances histamine-mediated wakefulness promotion. This mechanism increases arousal and reduces excessive daytime sleepiness in conditions characterized by hypersomnia.
Approved indications
- Narcolepsy type 1 and type 2
- Idiopathic hypersomnia
Common side effects
- Insomnia
- Headache
- Anxiety
- Nausea
- Irritability
Key clinical trials
- A Study of Pitolisant in Patients 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)
- 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)
- A Phase 3 Study to Assess the Safety and Efficacy of Pitolisant in Adult Patients With Idiopathic Hypersomnia (PHASE3)
- A Clinical Study to Evaluate the Efficacy and Safety of Pitolisant in the Treatment of EDS in Patients With OSA (PHASE3)
- Insulin Tolerance Test Study in Patients With Type 1 Diabetes (PHASE1)
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:
- Double-blind pitolisant CI brief — competitive landscape report
- Double-blind pitolisant updates RSS · CI watch RSS
- Harmony Biosciences Management, Inc. portfolio CI