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Wakix

(Pitolisant)

ApprovedSmall molecule

HHarmony Biosciences Management, Inc.

Pitolisant is used to treat narcolepsy, a sleep disorder that causes excessive daytime sleepiness and sudden loss of muscle control. It works by helping the brain stay more alert during the day.[2] Wakix is currently approved and in use across 5 indications[4].[1]

Wakix is one of 126 small molecule programs targeting Prader-Willi syndrome. It is currently in Approved.

5

Indications

20

Total Trials

5

Active Trials

125

Competitors

1

FDA Application

NDA/BLA/ANDA regulatory filings

3

Patents

Pipeline

Status by Indication[4][1]

Indication

Prader-Willi SyndromeRecruiting

Restless Legs SyndromeUnknown status

CognitionActive, not recruiting

Autism Spectrum DisorderActive, not recruiting

NarcolepsyUnknown

Prader-Willi SyndromePhase 3

Recruiting

Restless Legs SyndromeApproved

Unknown status

CognitionPreclinical

Active, not recruiting

Autism Spectrum DisorderPhase 2

Active, not recruiting

NarcolepsyPreclinical

Unknown

Competition

Competitive Landscape

85 competitors in Cognition

#DrugCompanyPhaseTrials

Lexapro

Elavil

Ketalar

AAultman Health Foundation

Precedex

MMcNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.

Wakix

HHarmony Biosciences Management, Inc.

Duragesic

Accutane

CConrado Roberto Hoffmann Filho

Namenda

Nuplazid

AACADIA Pharmaceuticals Inc.

Remimazolam Tosilate

FFujian Shengdi Pharmaceutical Co., Ltd.

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q2 2028

Phase 3Recruiting

Prader-Willi Syndrome

NCT06366464

Q3 2028

Phase 2Active, not recruiting

Prader-Willi Syndrome

NCT04257929

Q2 2030

Pregnancy

NCT05536011

Clinical

Trials by Indication[1]

Prader-Willi syndrome3 trials

A Study of Pitolisant in Participants With Prader-Willi Syndrome

Enrolling by invitation·HHarmony Biosciences Management, Inc.·Phase 3·NCT07219485·150 enrolled

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

Active, not recruiting·HHarmony Biosciences Management, Inc.·Phase 2·NCT04257929·65 enrolled

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

Recruiting·HHarmony Biosciences Management, Inc.·Phase 3·NCT06366464·134 enrolled

Cataplexy2 trials

Efficacy and Safety of Pitolisant in Pediatric Narcoleptic Patients With or Without Cataplexy, Double-blind Study Followed by a Prolonged Open-label Period

Completed·BBioprojet·Phase 3·NCT02611687·110 enrolled

Pitolisant to Assess Weekly Frequency of Cataplexy Attacks and EDS in Narcoleptic Patients (HARMONY CTP)

Completed·BBioprojet·Phase 3·NCT01800045·103 enrolled

Narcolepsy1 trial

Expanded Access Program to Provide Treatment With Pitolisant to Adult Patients in the U.S. With Excessive Daytime Sleepiness Associated With Narcolepsy With or Without Cataplexy

No longer available·HHarmony Biosciences Management, Inc.··NCT03433131

Pregnancy1 trial

WAKIX® (Pitolisant) Pregnancy Registry

Recruiting·HHarmony Biosciences Management, Inc.··NCT05536011·1,329 enrolled

Heavy Drinker1 trial

Pitolisant Effects on Alcohol Self-Administration in Heavy Drinkers

Terminated·—·Phase 1·NCT04596267·9 enrolled

12 more indications

Idiopathic Hypersomnia1 trial

A Long-Term Safety and Effectiveness Study to Evaluate Pitolisant in Adult Patients With Idiopathic Hypersomnia

Completed·HHarmony Biosciences Management, Inc.·Phase 3·NCT05458128·119 enrolled

Abuse Potential1 trial

Determination the Abuse Potential of Pitolisant in Healthy, Non-Dependent Recreational Stimulant Users

Completed·BBioprojet·Phase 1·NCT03152123·43 enrolled

Cognition1 trial

Pitolisant Effects on Affect and Cognition Exploratory Study (PEACE Study)

Completed·UUniversity of Oxford·N/A·NCT05849675·58 enrolled

Alcohol Use Disorder1 trial

A Trial Evaluating Pitolisant (BF2.649) in Alcohol Use Disorder Treatment

Withdrawn·BBioprojet·Phase 2·NCT02800083

Excessive Daytime Sleepiness1 trial

Pitolisant (BF2.649) in the Treatment of Excessive Daytime Sleepiness in Patients With Obstructive Sleep Apnoea Syndrome, Treated or Not by Nasal Continuous Positive Airway Pressure, But Still Complaining of Excessive Daytime Sleepiness

Withdrawn·BBioprojet·Phase 3·NCT02978651

Treatment1 trial

Pitolisant (BF2.649) in the Treatment of EDS in Patients With OSA

Completed·BBioprojet·Phase 3·NCT02739568·389 enrolled

Type 1 Diabetes1 trial

Insulin Tolerance Test Study in Patients With Type 1 Diabetes

Terminated·HHigh Point Clinical Trials Center·Phase 1·NCT04026750·5 enrolled

Absorption1 trial

Study to Assess the Absorption, Distribution, Metabolism and Excretion (ADME) of [14C]-Pitolisant in Healthy Male Volunteers

Completed·BBioprojet·Phase 1·NCT02929342·8 enrolled

Autism Spectrum Disorder1 trial

Proof of Concept Study on Pitolisant Effect on Autism Spectrum Disorders in Children and Adolescents

Active, not recruiting·BBioprojet·Phase 2·NCT05953389·62 enrolled

Efficacy and Safety1 trial

A Clinical Study to Evaluate the Efficacy and Safety of Pitolisant in the Treatment of EDS in Patients With OSA

Completed·CCitrine Medicine Limited·Phase 3·NCT05223166·240 enrolled

Restless Legs Syndrome1 trial

Pitolisant in Refractory Restless Legs Syndrome

Unknown status·WWilliam Ondo, MD·Phase 4·NCT05581576·18 enrolled

Myotonic Dystrophy 11 trial

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

Completed·HHarmony Biosciences Management, Inc.·Phase 2·NCT04886518·30 enrolled

Regulatory

FDA Approval Timeline[5]

Aug 14, 2019

WAKIX

NDA NDA211150

Sources

References

[1]
ClinicalTrials.gov — 20 trial records for Pitolisant. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Wakix drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — Pitolisant compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for Pitolisant. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (NDA NDA211150). U.S. Food & Drug Administration.
[6]
FDA Orange Book — Patent and exclusivity data for Wakix. U.S. Food & Drug Administration.
[7]
SEC EDGAR — Harmony Biosciences Management, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Histamine H3 Receptor Inverse Agonist[3]
Modality: Small molecule[3]
Highest Phase: Approved[1]
Company: HHarmony Biosciences Management, Inc.

Patent Landscape[6]

Patent Cliff

Sep 26, 2029

Earliest patent expiration — generic entry possible after this date

Patent #Expires

8354430Feb 6, 2026

8486947Sep 26, 2029

8207197Mar 7, 2030

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