Back to discover
Drug

Pitolisant

Established medicine
ClassHistamine H3 receptor inverse agonistResearchOngoing clinical research

Also known as Wakix.

RxNorm2197878ChEMBLCHEMBL462605ATCN07XX11UNII4BC83L4PIY
3
Active clinical trials
Histamine H3 receptor
Primary target
4
Regulatory events
Latest activity
beta

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

Viatris’ Wakix receives Japanese approval to treat OSAS and narcolepsy

Regulatory2026-09-17Regulatory news · Pharmaceutical Technology

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 trial2026-08-25Results expected Q2 2027 · ClinicalTrials.gov

Approval: PITOLISANT (ANDA218846)

Regulatory2026-08-14FDA

Approval: PITOLISANT (ANDA218873)

Regulatory2026-08-14FDA

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 trial2025-10-31Primary 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

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
ATC codes
N07XX11
UNII
4BC83L4PIY
Primary mechanism
Histamine H3 receptor inverse agonist
Regulatory jurisdictions
emafda

Pharmacology & targets

1 target

Known molecular targets and mechanisms supported by curated pharmacology databases.

Histamine H3 receptor
HRH3 · Q9Y5N1
INVERSE AGONISTHistamine H3 receptor inverse agonist

Regulatory timeline

4 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
2026-08-14
Authorities
FDA · EMA
Total events
4
fdaU.S. Food and Drug Administration· 2 events
2026-08-14Approval
Approval: PITOLISANT (ANDA218846)
Evidence ↗
2026-08-14Approval
Approval: PITOLISANT (ANDA218873)
Evidence ↗
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 ↗

Data from the U.S. Food and Drug Administration (U.S. Food and Drug Administration), public domain (CC0).

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

Clinical trials

18 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
18
registered trials across all phases
LATEST COMPLETION 2026
3
Active studies
2
Recruiting
10
Late-stage (III+)
10
Completed
5
Discontinued
PHASE DISTRIBUTIONn = 18
Phase 14Phase 24Phase 38Phase 41Phase N / A1

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

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

Unknown statusPH IVWilliam Ondo, MDn=182023

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

Enrolling by invitationPH IIIHarmony Biosciences Management, Inc.n=1502030

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

RecruitingPH IIIHarmony Biosciences Management, Inc.n=1342028

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

CompletedPH IIIHarmony Biosciences Management, Inc.n=1192025

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

CompletedPH IIIBioprojetn=1102025

Recent completions

Trials that read out recently, adding to the completed evidence base.

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

CompletedPH IIHarmony Biosciences Management, Inc.n=652026

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

CompletedPH IIIHarmony Biosciences Management, Inc.n=1192025

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

CompletedPH IIIBioprojetn=1102025

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

CompletedPH IIHarmony Biosciences Management, Inc.n=302024

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

CompletedN/AUniversity of Oxfordn=582024

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

CompletedPH IIICitrine Medicine Limitedn=2402023
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.