Pitolisant clinical trials
Pitolisant is named as an intervention in 6 current clinical trials in our USA, Canada, France, Spain, Italy, UK, Germany, Belgium, Sweden, Denmark, Poland and Australia registers. 4 are recruiting patients now. The most common phase is Phase 3. The sponsor with the most trials is Harmony Biosciences Management, Inc (5). The most-studied condition is Prader-Willi Syndrome. The EU clinical trials register (CTIS) separately lists 3 authorised or ongoing trials.
Where Pitolisant is approved
All regulators →Of the three regulators tracked here, the first to approve Pitolisant was the EU, on 31 Mar 2016. It is approved in 3 of the 3 regulators tracked here.
From the regulators' own registers: FDA Drugs@FDA, the EMA medicines list and Health Canada's Notice of Compliance database. Dates are the first listed approval of a product containing only this substance; combination products and nationally authorised EU medicines are not counted.
Pitolisant patents and exclusivity
Expiry tracker →Protections still running for marketed products containing Pitolisant, as listed by the regulators. Expired patents and exclusivities, and discontinued products, are not shown.
- 2 listed patents; the last listed expires 7 Mar 2030 (US 8207197).
- Latest patent on the drug substance itself: 7 Mar 2030 (US 8207197).
- Exclusivity ODE-519: Orphan drug exclusivity: treatment of cataplexy in pediatric patients 6 years of age and older with narcolepsy, until 13 Feb 2033.
- Exclusivity ODE-489: Orphan drug exclusivity: treatment of excessive daytime sleepiness (eds) in pediatric patients 6 years of age and older with narcolepsy, until 21 Jun 2031.
- Exclusivity ODE-331: Orphan drug exclusivity: treatment of cataplexy in adult patients with nacrolepsy, until 13 Oct 2027.
- Exclusivity NPP: New patient population, until 21 Jun 2027.
- Wakix for treatment of narcolepsy: until 4 Apr 2028 (includes 24 months' paediatric extension). Union Register
Reproduced from the FDA Orange Book and Purple Book (checked 8 Nov 2026) and the European Commission's Union Register of medicinal products (CC BY 4.0; the EU expiry date is calculated exactly as the Commission's register displays it). Listed patents and dates are as submitted to or published by the regulator and may not reflect litigation, invalidation, settlements, lapsed fees or later extensions. This is not legal advice or a freedom-to-operate opinion, and it does not say when a generic or biosimilar will launch.
What health systems spend on Pitolisant
All medicines →From NHSBSA Prescription Cost Analysis (Open Government Licence v3.0) and CMS State Drug Utilization Data. Both are gross amounts: neither system publishes its discounts or rebates per drug. Products combining several substances are not counted here.
How Pitolisant works
All drug targets →- Histamine H3 receptor inverse agonist Inverse agonistTarget: HRH3 histamine receptor H3✓ In ChEMBL and the Guide to Pharmacology
- CataplexyPhase 3
- Parkinson diseasePhase 3
- Prader-Willi syndromePhase 3
- Idiopathic hypersomniaPhase 3
- SchizophreniaPhase 3
- Autism spectrum disorderPhase 2
- Myotonic dystrophy type 1Phase 2
- Schizoaffective disorderPhase 2
- Alcohol dependencePhase 1
- Type 1 diabetes mellitusPhase 1
Mechanisms and diseases from ChEMBL via the Open Targets Platform (release 26.09), ChEMBL record CHEMBL462605, CC BY-SA 3.0. Targets checked against the IUPHAR/BPS Guide to PHARMACOLOGY (2026.3.1), ligand 8924, CC BY-SA 4.0. Trial phases are not approvals: approved uses are shown from the regulators' own records where we hold them.
NICE appraisals of Pitolisant
All NICE appraisals →NICE, which decides whether the NHS in England should fund new medicines, has published 1 technology appraisal of Pitolisant. 1 is current.
| Appraisal | NICE recommendation | Published | Status |
|---|---|---|---|
| Pitolisant hydrochloride for treating excessive daytime sleepiness caused by obstructive sleep apnoea NICE TA776 | Not recommended | 9 Mar 2022 | Current |
NICE decisions apply to the NHS in England. In Ireland the HSE decides on reimbursement, advised by the National Centre for Pharmacoeconomics. Titles, categories and dates link to the guidance on nice.org.uk. © NICE 2026 technology appraisal guidance. Available from www.nice.org.uk/guidance. All rights reserved. Subject to Notice of rights. NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.
Patient leaflets for Pitolisant
The package leaflet is the official guide for patients that comes with every medicine. These links go to the regulators that publish it.
- IEIreland: 4 products on the HPRA listEach product record links to the HPRA, which publishes the leaflet and SmPC.
- EU
- UKUnited Kingdom: Search MHRA ProductsPatient information leaflets and SmPCs for UK-licensed products.
- USUnited States: Search DailyMedFDA-approved labels, including patient and medication guides.
Leaflets differ between brands and countries; always read the one that comes with your own medicine. This is regulatory information, not medical advice.
Reference identifiers for Pitolisant
- ATC code
- N07XX11
- ChEMBL
- CHEMBL462605
- DrugBank
- DB11642
- PubChem CID
- 9948102
- CAS number
- 362665-56-3
- FDA UNII
- 4BC83L4PIY
- Wikidata
- Q426044
- Wikipedia
- Pitolisant
Codes that identify this medicine in other databases, from Wikidata (CC0), matched by its ChEMBL ID. The ATC code is the WHO classification of what the medicine is used for.
Trials by status
Trials by phase
Clinical trials of Pitolisant
6 trialsRegisters list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Trial titles and statuses are copied from the registry as published.
Pitolisant in the EU trials register (CTIS)
3 trialsAuthorised or ongoing trials in the EU Clinical Trials Information System that list a product containing Pitolisant. Many trials are registered in both CTIS and ClinicalTrials.gov, so these are shown separately rather than added to the count above. Trials with a site in Ireland are listed first.
| Study | Phase | Status | Sponsor | Countries |
|---|---|---|---|---|
|
A Phase 3, Randomized, Double-blind, Placebo-Controlled, Efficacy and Safety Study of HBS-301 in Participants with Idiopathic Hypersomnia (IH) Followed by an Open-label Extension
2025-523822-42-00 · CTIS ↗ · Nervous System Diseases
|
Phase 3 | Authorised | Harmony Biosciences Management Inc | France, Germany, Belgium + 3 more |
|
A Phase 3, Randomized, Double-blind, Placebo-Controlled, Efficacy and Safety Study of HBS-301 in Participants with Narcolepsy Followed by an Open-label Extension
2025-523821-17-00 · Nervous System Diseases
|
Phase 3 | Ongoing | Harmony Biosciences Management Inc | Belgium, Italy, Germany + 3 more |
|
The headache- and migraine-inducing effects of pitolisant in women with migraine without aura
2026-525521-20-00 · CTIS ↗ · Nervous System Diseases
|
Phase 1 | Authorised | Syddansk Universitet (University of Southern Denmark) | Denmark |
Pitolisant across our data
Ireland · EU · USThe same active substance in our other registers, matched by its exact name. Each entry links to the register record it comes from.
Licensed in Ireland (HPRA)
4 single-substance products containing Pitolisant on the HPRA medicines list.
- Ozawade 18 mg film-coated tabletsBioprojet Pharma · EU/1/21/1546/002-003
- Ozawade 4.5 mg film-coated tabletsBioprojet Pharma · EU/1/21/1546/001
- Wakix 18 mg film-coated tabletsBioprojet Pharma · EU/1/15/1068/002
- Wakix 4.5 mg film-coated tabletsBioprojet Pharma · EU/1/15/1068/001
Supply shortages
No entry for Pitolisant in the EMA, FDA or German BfArM shortage lists we track.
Conditions studied with Pitolisant
8 conditionsSponsors
lead sponsor per trial| Lead sponsor | Trials |
|---|---|
| Harmony Biosciences Management, Inc | 5 |
| Bioprojet | 1 |
By country register
A trial with sites in more than one country appears in each of those registers. CTIS does not publish a recruiting flag; its trials may also appear in the registers above.
Related compounds
Questions about Pitolisant trials
How many clinical trials are testing Pitolisant?
6 current trials in our USA, Canada, France, Spain, Italy, UK, Germany, Belgium, Sweden, Denmark, Poland and Australia registers name Pitolisant (also registered as HBS-301) as a drug or biological intervention, as of the registry sync on 9 November 2026. Completed and withdrawn studies are not counted.
Are Pitolisant trials recruiting patients?
Yes. 4 of the 6 trials are recruiting: 4 with sites in USA, 3 with sites in Canada, 1 with sites in France, 1 with sites in Spain, 1 with sites in Italy, 1 with sites in UK, 1 with sites in Germany, 1 with sites in Belgium, 1 with sites in Sweden, 1 with sites in Denmark, 1 with sites in Poland and 1 with sites in Australia. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.
Who sponsors Pitolisant clinical trials?
Harmony Biosciences Management, Inc (5) and Bioprojet (1). The number in brackets is how many trials each organisation leads.
Is Pitolisant licensed in Ireland?
Yes. The HPRA medicines list includes 4 single-substance products containing Pitolisant, for example Ozawade 18 mg film-coated tablets (Bioprojet Pharma). A licence does not mean a product is currently marketed or reimbursed; see each product's record.
Is there a Pitolisant shortage?
Not in the lists we track: Pitolisant has no entry in the EMA, FDA or German BfArM shortage registers as of their latest sync.
Where does this information come from?
Every trial, status, phase and sponsor on this page is copied from public ClinicalTrials.gov records by a daily sync. Nothing here describes how Pitolisant works or whether it is effective, and it is not medical advice.
How this page is built
Everything on this page is copied from public ClinicalTrials.gov registry records — it is not written from memory, and nothing here describes how Pitolisant works or whether it is effective. Each trial links to its own record so you can check it yourself.
- ClinicalTrials.gov (U.S. National Library of Medicine) ↗ Trial IDs, titles, intervention names and synonyms, phase, overall status, lead sponsor and conditions for every trial on this page
This page is built automatically: a daily sync job reads the registry records behind our country trial registers, collects every trial that lists Pitolisant (or a registered synonym) as a drug or biological intervention, and tabulates the registry fields directly. Placebos and generic comparators are excluded. There is no AI drafting and no per-compound editorial pass, and the page has not been clinically reviewed. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The ClinicalTrials.gov data on this page was last retrieved on 9 Nov 2026. How every register is built: methodology · fixes we have made: corrections log.