Drug

Pitolisant

Approved · EMA

Late-stage development

Also known as Wakix.

RxNorm2197878 ChEMBLCHEMBL462605UNII4BC83L4PIY

Active clinical trials

Regulatory events

Latest activity

beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Phase 3, Randomized, Double-Blind, Placebo-controlled, Efficacy and Safety Study of Pitolisant Followed by an Open-Label Extension in Patients With Prader-Willi Syndrome

Clinical trial2026-07-01Primary completion · ClinicalTrials.gov

A Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate the Safety and Efficacy of Pitolisant in Patients With Prader-Willi Syndrome, Followed by an Open Label Extension

Clinical trial2026-05-12Results posted · ClinicalTrials.gov

An Open-Label Study to Evaluate Safety of Pitolisant in Participants With Prader-Willi Syndrome

Clinical trial2026-03-17Results expected Q3 2030 · ClinicalTrials.gov

An Exploratory, Multicenter, Randomized, Double-blind, Placebo-controlled Study Evaluating the Effect and Safety of Pitolisant in Children and Adolescents With Autism Spectrum Disorders

Clinical trial2025-10-01Primary completion · ClinicalTrials.gov

An Open-Label Study to Evaluate the Long-Term Safety and Effectiveness of Pitolisant in Adult Patients With Idiopathic Hypersomnia Who Completed Study HBS-101-CL-010

Clinical trial2025-10-01Completed · ClinicalTrials.gov

Double Blind, Multicentre, Randomized, Placebo-controlled Trial to Evaluate Safety and Efficacy of Pitolisant in Children From 6 to Less Than 18 Years With Narcolepsy With/Without Cataplexy, Followed by a Prolonged Open-label Period

Clinical trial2025-06-26Results posted · ClinicalTrials.gov

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1, Followed by an Open-Label Extension

Clinical trial2025-04-25Results posted · ClinicalTrials.gov

An fMRI Investigation of the Effects of Selective Histamine-3 Receptor Antagonism on Cognitive and Emotional Processing in Healthy Individuals

Clinical trial2024-05-08Completed · ClinicalTrials.gov

A Randomized, Double-blind, Placebo-controlled, Multicenter Clinical Study to Evaluate the Efficacy and Safety of Pitolisant in the Treatment of Excessive Daytime Sleepiness in Patients With Obstructive Sleep Apnea

Clinical trial2023-07-18Completed · ClinicalTrials.gov

Open Label Assessment of Pitolisant (Wakix) as Adjunct Therapy for Patients With Refractory Restless Legs Syndrome

Clinical trial2023-05-01Primary completion · ClinicalTrials.gov

Approval: Ozawade (EMA)

Regulatory2021-09-01EMA

Approval: Wakix (EMA)

Regulatory2016-03-31EMA

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name

Pitolisant

Aliases & brands

Wakix

RxNorm CUI

2197878

ChEMBL ID

CHEMBL462605

UNII

4BC83L4PIY

Regulatory jurisdictions

ema

Regulatory timeline

2 events

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval

2016-03-31

Latest approval

2021-09-01

Authorities

EMA

Total events

emaEuropean Medicines Agency· 2 events

2021-09-01Approval

Approval: Ozawade (EMA)

Indication: Ozawade is indicated to improve wakefulness and reduce excessive daytime sleepiness (EDS) in adult patients with obstructive sleep apnoea (OSA)

Evidence ↗

2016-03-31Approval

Approval: Wakix (EMA)

Indication: Wakix is indicated in adults, adolescents and children from the age of 6 years for the treatment of narcolepsy with or without cataplexy (see also section 5.1).

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.