Product Description
Pitolisant is used to treat excessive daytime sleepiness caused by narcolepsy (a condition that causes excessive daytime sleepiness) and to treat cataplexy (episodes of muscle weakness that begin suddenly and last for a short time) in adults with narcolepsy. Pitolisant is in a class of medications called H3 blockers. It works by changing the amounts of certain natural substances in the area of the brain that controls sleep and wakefulness. (Sourced from: https://medlineplus.gov/druginfo/meds/a619055.html)
Mechanisms of Action: H3 Antagonist
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: Orphan Drug - Orphan Drug - Prader-Willi SyndromePriority Review - Narcolepsy *
Approval Status: Approved
Approved Countries: Austria | Belgium | Canada | Croatia | Czech | Denmark | Estonia | European Medicines Agency | Finland | France | Germany | Greece | Hong Kong | Hungary | Iceland | Ireland | Israel | Italy | Korea | Latvia | Lithuania | Luxembourg | Netherlands | Norway | Poland | Portugal | Romania | Slovakia | Slovenia | Spain | Sweden | Switzerland | Taiwan | United Kingdom | United States
Approved Indications: Cataplexy | Narcolepsy
Known Adverse Events: Insomnia
Company: Harmony Biosciences, LLC
Company Location: PLYMOUTH MEETING PA 19462
Company CEO: John C. Jacobs
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Canada, China, France, Italy, Japan, Spain, United States
Active Clinical Trial Count: 13
Highest Development Phases
Phase 3: Disorders of Excessive Somnolence|Narcolepsy|Prader-Willi Syndrome|Sleep Apnea, Obstructive
Phase 2: Autism Spectrum Disorder|Myotonic Disorders|Myotonic Dystrophy Type 1
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| HBS-101-CL-312 | P3 |
Recruiting |
Prader-Willi Syndrome |
2026-07-01 |
|
| HBS-101-CL-011 | P3 |
Active, not recruiting |
Disorders of Excessive Somnolence |
2025-08-01 |
|
| P21-01 | P2 |
Unknown Status |
Autism Spectrum Disorder |
2025-07-30 |
|
| P11-06 | P3 |
Unknown Status |
Narcolepsy |
2025-03-31 |