Drugs / Pitolisant
Pitolisant
also known as
BF2.649 · Wakix · BF-2649 HYDROCHLORIDE · Ciproxidine · HBS-101 · Ozawade · PITOLISANT HYDROCHLORIDE · Tiprolisant · +1 more
Small molecule targets HRH3
Regulatory milestones approvals, filings & regulatory actions · 7 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed idiopathic hypersomnia 2 milestones | |||||
| Rejected (CRL) | US (FDA) | — | idiopathic hypersomnia | 2025-02-19 | “Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) for pitolisant” harmonybiosciences.com ↗ |
| Filed | US (FDA) | — | idiopathic hypersomnia | — | “FDA decision on file acceptance of pitolisant sNDA for Idiopathic Hypersomnia (IH) IH sNDA for pitolisant submitted in Q4 2024” harmonybiosciences.com ↗ |
| Filed narcolepsy 1 milestone | |||||
| Filed | US (FDA) | — | narcolepsy | — | “Pitolisant GR (Gastro-resistant) Tablets: On track to launch in 1H 2027 NDA accepted by FDA in July” harmonybiosciences.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansions (2) | US (FDA) | WAKIX | — | 2024-06-21 – 2026-02-13 | fda.gov ↗ |
| Approved | US (FDA) | WAKIX | — | 2019-08-14 | fda.gov ↗ |
| Approved | EU (EMA) | Wakix | — | 2016-03-31 | europa.eu ↗ |
Trials 31
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 16 trials | ||||||
| Phase 1 | NCT07860736 | Jun 2026 | — | Harmony Biosciences Management, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT04596267 | Sep 2021 → Feb 2023 | alcohol-related disorders | Boston Medical Center | Terminated | No outcome recorded Stop: Enrollment |
| Phase 1 | NCT04026750 | Sep 2019 → Feb 2020 | hypoglycemia, type 1 diabetes mellitus | High Point Clinical Trials Center | Terminated | No outcome recorded |
| Phase 1 | NCT03152123 | Mar → Oct 2017 | substance abuse | Bioprojet | Completed | No outcome recorded |
| Phase 1 | NCT02929342 | Jul → Aug 2016 | — | Bioprojet | Completed | No outcome recorded |
| Phase 1 | NCT01619033 | Jul 2011 → Nov 2012 | impaired renal function disease | Bioprojet | Completed | No outcome recorded |
| Phase 25 trials | ||||||
| Phase 2 | NCT05953389 | Dec 2023 → Oct 2025 overdue | autism spectrum disorder | Bioprojet | Active not recruiting | No outcome recorded |
| Phase 2 | NCT04257929 | Dec 2020 → Aug 2022 | Prader-Willi syndrome | Harmony Biosciences Management, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT02800083 | Oct 2016 → Dec 2019 | alcohol abuse | Bioprojet | Withdrawn | No outcome recorded Stop: Business |
| Phase 2 | NCT01620554 | Oct 2010 → Dec 2011 | hypersomnia, obstructive sleep apnea syndrome | Bioprojet | Completed | No outcome recorded |
| Phase 2 | NCT00642928 | Oct 2007 → Mar 2009 | Parkinson disease, hypersomnia | Bioprojet | Completed | No outcome recorded |
| Phase 317 trials · 4 met primary | ||||||
| Phase 3 | NCT07219485 | Aug 2025 → Aug 2030 expected | Prader-Willi syndrome | Harmony Biosciences Management, Inc. | Enrolling by invitation | No outcome recorded |
| Phase 3 | NCT06366464 | May 2024 → Jul 2026 overdue | Prader-Willi syndrome | Harmony Biosciences Management, Inc. | Recruiting | No outcome recorded |
| Phase 3 | NCT05458128 | Aug 2022 → Oct 2025 | idiopathic hypersomnia | Harmony Biosciences Management, Inc. | Completed | No outcome recorded |
| Phase 3 | NCT05223166 | Apr 2022 → Jul 2023 | sleep-wake disorder | Citrine Medicine Limited | Completed | No outcome recorded |
| Phase 3 | NCT02611687 | Jun 2016 → Apr 2021 | narcolepsy without cataplexy, narcolepsy-cataplexy syndrome | Bioprojet | Completed | Met primary |
| Phase 3 | NCT02739568 | Apr 2016 → Apr 2020 | hypersomnia, obstructive sleep apnea syndrome | Bioprojet | Completed | No outcome recorded |
| Phase 3 | NCT01800045 | Apr 2013 → Jan 2015 | narcolepsy-cataplexy syndrome | Bioprojet | Completed | Met primary |
| Phase 3 | NCT01789398 | Sep 2012 → Aug 2014 | narcolepsy | Bioprojet | Completed | No outcome recorded |
| Phase 3 | NCT01072968 | Oct 2011 → May 2014 | obstructive sleep apnea syndrome | Bioprojet | Completed | Met primary |
| Phase 3 | NCT01071876 | Aug 2011 → Mar 2014 | hypersomnia, obstructive sleep apnea syndrome | Bioprojet | Completed | Met primary |
| Phase 3 | NCT01399606 | May 2011 → Oct 2013 | narcolepsy-cataplexy syndrome | Bioprojet | Completed | No outcome recorded |
| Phase 3 | NCT01638403 | Nov 2010 → Apr 2012 | narcolepsy | Bioprojet | Completed | No outcome recorded |
| Phase 3 | NCT01066442 | Mar 2010 → Aug 2012 | Parkinson disease | Bioprojet | Completed | No outcome recorded |
| Phase 3 | NCT01036139 | Dec 2009 → Aug 2012 | Parkinson disease | Bioprojet | Completed | No outcome recorded |
| Phase 3 | NCT01067235 | Oct 2009 → Jul 2010 | narcolepsy-cataplexy syndrome | Bioprojet | Completed | No outcome recorded |
| Phase 3 | NCT01067222 | May 2009 → Jul 2010 | hypersomnia, narcolepsy-cataplexy syndrome | Bioprojet | Completed | Mixed |
| Phase 3 | NCT02978651 | ? → Jan 2017 | hypersomnia, obstructive sleep apnea syndrome | Bioprojet | Withdrawn | No outcome recorded Stop: Business |
| Phase 41 trial | ||||||
| Phase 4 | NCT05581576 | Sep 2022 → May 2023 | restless legs syndrome | William Ondo, MD | Unknown | No outcome recorded |
| Phase not applicable1 trial | ||||||
| — | NCT05849675 | Apr 2023 → Feb 2024 | atypical depressive disorder, cognitive disorder | University of Oxford | Completed | No outcome recorded |
| Phase not stated1 trial | ||||||
| — | NCT03433131 | — | narcolepsy without cataplexy | Harmony Biosciences Management, Inc. | No longer available | No outcome recorded |
| Comparator or background therapy3 trials | ||||||
| Phase 2 | NCT04886518 Comparator | Jun 2021 → Oct 2023 | hypersomnia, myotonic dystrophy type 1 | Harmony Biosciences Management, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT00690274 Comparator | Jun 2008 → Oct 2011 | schizophrenia | University of Texas Southwestern Medical Center | Completed | No outcome recorded |
| Phase 3 | NCT05156047 Comparator | May 2022 → Sep 2023 | idiopathic hypersomnia | Harmony Biosciences Management, Inc. | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 23
| Date | Issuer | Release |
|---|---|---|
| 2026-09-16 | Pfizer's Upjohn has merged with Mylan to form Viatris Inc. | Regulatory Viatris Announces Approval of WAKIX® (Pitolisant) in Japan, a First-in-Class Treatment for Narcolepsy and Excessive Daytime Sleepiness Associated With Obstructive Sleep Apnea Syndrome viatris.com ↗
WAKIX is the first histamine H3 receptor antagonist/inverse agonist approved in Japan for these indications. |
| 2026-08-17 | Lupin Limited | Regulatory Lupin Announces Approval from U.S. FDA for Pitolisant Tablets lupin.com ↗
Mumbai, Naples, August 17, 2026: Global pharma major Lupin Limited (Lupin) today announced that it has received the final approval from the United States Food and Drug Administration (U.S. FDA) for its Abbreviated New Drug Application for Pitolisant Tablets, 4.45 mg and 17.8 mg. |
| 2026-03-25 | Lupin Limited | Regulatory Lupin Receives Tentative Approval from U.S. FDA for Pitolisant Tablets lupin.com ↗
The U.S. FDA has tentatively approved Lupin’s Pitolisant Tablets 4.45 mg and 17.8 mg as bioequivalent to Wakix® for the indication in the approved labeling. |
| 2026-02-17 | Harmony Biosciences Management, Inc. | Regulatory Harmony Biosciences Receives U.S. Food and Drug Administration Approval for WAKIX® (pitolisant) for the Treatment of Cataplexy in Pediatric Narcolepsy harmonybiosciences.com ↗
WAKIX is now the first-and-only FDA-approved non-scheduled treatment for pediatric and adult narcolepsy patients with or without cataplexy. |
| 2025-11-24 | Harmony Biosciences Management, Inc. | Results Harmony Biosciences Announces Positive Results From the Pivotal Bioequivalence Study for Pitolisant Gastro-Resistant Formulation harmonybiosciences.com ↗
The pivotal BE study confirmed that 17.8mg of pitolisant GR is bioequivalent to existing 17.8mg WAKIX® tablets. |
| 2025-02-19 | Harmony Biosciences Management, Inc. | Regulatory Harmony Biosciences Provides Update on the Status of the Supplemental New Drug Application for Pitolisant in Idiopathic Hypersomnia harmonybiosciences.com ↗
Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) today announced that it received a Refusal to File (RTF) letter from the U.S. Food and Drug Administration (FDA) for pitolisant for the treatment of excessive daytime sleepiness (EDS) in adult patients with idiopathic hypersomnia (IH). |
| 2024-10-01 | Harmony Biosciences Management, Inc. | Results HARMONY BIOSCIENCES HIGHLIGHTS NEW DATA, ROBUST LATE-STAGE PIPELINE WITH NEAR-TERM VALUE CREATION OPPORTUNITIES AND ITS BOLD NEW VISION AT INVESTOR DAY harmonybiosciences.com ↗
New Data Shows Clinically Meaningful Sustained Efficacy of Pitolisant in Patients with Idiopathic Hypersomnia in Long-Term Extension Study |
| 2024-06-25 | Harmony Biosciences Management, Inc. | Regulatory HARMONY BIOSCIENCES ACKNOWLEDGES U.S. FOOD & DRUG ADMINISTRATION (FDA) ACTION DENYING THE CITIZEN PETITION FOR WAKIX® (PITOLISANT) harmonybiosciences.com ↗
today announced that the U.S. Food and Drug Administration denied the Citizen Petition filed by a short seller claiming that WAKIX is not safe and effective for the treatment of excessive daytime sleepiness (EDS) and cataplexy in adults with narcolepsy. |
| 2024-06-24 | Harmony Biosciences Management, Inc. | Regulatory HARMONY BIOSCIENCES RECEIVES U.S. FOOD AND DRUG ADMINISTRATION APPROVAL FOR WAKIX® (PITOLISANT) IN PEDIATRIC PATIENTS WITH NARCOLEPSY harmonybiosciences.com ↗ |
| 2024-06-05 | Harmony Biosciences Management, Inc. | Results HARMONY BIOSCIENCES PRESENTS POSITIVE DATA FOR PITOLISANT IN THE TREATMENT OF EXCESSIVE DAYTIME SLEEPINESS AND FATIGUE IN MYOTONIC DYSTROPHY TYPE 1 harmonybiosciences.com ↗
Harmony Biosciences (Nasdaq: HRMY) presented data from its Phase 2 signal detection study showing that pitolisant reduced excessive daytime sleepiness (EDS) and fatigue in adults with Myotonic dystrophy Type 1 (DM1). |
| 2024-04-30 | Harmony Biosciences Management, Inc. | Results Harmony Biosciences Reports Strong First Quarter Financial Results and Acceleration of Its Growth Strategy; Advances Pitolisant Franchise to Extend Revenue Potential Beyond 2040; Strengthens Sleep/Wake Leadership and Diversifies Into Rare Epilepsy harmonybiosciences.com ↗
Reports Positive Pharmacokinetic Data on Next-Generation Formulation 1 (NG1); |
| 2024-02-21 | Harmony Biosciences Management, Inc. | Regulatory U.S. FOOD AND DRUG ADMINISTRATION GRANTS PRIORITY REVIEW TO HARMONY BIOSCIENCES' APPLICATION FOR WAKIX® (PITOLISANT) IN PEDIATRIC NARCOLEPSY harmonybiosciences.com ↗ |
| 2024-02-20 | Harmony Biosciences Management, Inc. | Regulatory HARMONY BIOSCIENCES RECEIVES U.S. FOOD AND DRUG ADMINISTRATION ORPHAN DRUG DESIGNATION FOR PITOLISANT IN PRADER-WILLI SYNDROME harmonybiosciences.com ↗
Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to pitolisant for the treatment of Prader-Willi syndrome (PWS). |
| 2023-12-07 | Harmony Biosciences Management, Inc. | Results HARMONY BIOSCIENCES ANNOUNCES POSITIVE TOPLINE DATA FROM PHASE 2 SIGNAL DETECTION STUDY EVALUATING PITOLISANT IN ADULT PATIENTS WITH MYOTONIC DYSTROPHY TYPE 1 harmonybiosciences.com ↗
Harmony Biosciences Holdings, Inc. ("Harmony" or the "Company") (Nasdaq: HRMY) today announced positive topline results from its Phase 2 signal detection study evaluating the safety and efficacy of pitolisant in adult patients with myotonic dystrophy type 1 (DM1). |
| 2023-10-13 | Harmony Biosciences Management, Inc. | Results HARMONY BIOSCIENCES ANNOUNCES TOPLINE DATA FROM PHASE 3 INTUNE STUDY EVALUATING PITOLISANT IN PATIENTS WITH IDIOPATHIC HYPERSOMNIA harmonybiosciences.com ↗
Although primary outcome for excessive daytime sleepiness (EDS) between pitolisant and placebo did not reach statistical significance in the randomized withdrawal phase, further data analysis is ongoing to inform next steps |
| 2023-10-05 | Harmony Biosciences Management, Inc. | Results HARMONY BIOSCIENCES PRESENTS NEW SECONDARY OUTCOME DATA FROM PHASE 2 SIGNAL DETECTION STUDY IN PATIENTS WITH PRADER-WILLI SYNDROME harmonybiosciences.com ↗
The poster presentation of secondary outcomes data included improvements in behavioral symptoms (as measured by the Aberrant Behavioral Checklist-2), especially in the higher-dose pitolisant group. |
| 2023-09-07 | Harmony Biosciences Management, Inc. | Regulatory HARMONY BIOSCIENCES ANNOUNCES US FOOD & DRUG ADMINISTRATION ORPHAN DRUG DESIGNATION FOR PITOLISANT FOR TREATMENT OF IDIOPATHIC HYPERSOMNIA harmonybiosciences.com ↗ |
| 2023-06-06 | Harmony Biosciences Management, Inc. | Results HARMONY BIOSCIENCES ANNOUNCES POSITIVE PHASE 2 SIGNAL DETECTION STUDY EVALUATING PITOLISANT FOR EXCESSIVE DAYTIME SLEEPINESS IN PRADER-WILLI SYNDROME AT SLEEP 2023 harmonybiosciences.com ↗
Notably, both the high- and low-dose pitolisant treatment arms demonstrated greater mean improvement from baseline in the Epworth Sleepiness Scale for Children and Adolescents (ESS- CHAD , Parent / Caregiver version) scores in the overall patient population when compared to placebo. |
| 2022-11-01 | Harmony Biosciences Management, Inc. | Results HARMONY BIOSCIENCES ANNOUNCES TOPLINE RESULTS FROM PHASE 2 PROOF-OF-CONCEPT STUDY EVALUATING PITOLISANT FOR EXCESSIVE DAYTIME SLEEPINESS IN PATIENTS WITH PRADER-WILLI SYNDROME harmonybiosciences.com ↗
Topline data show positive signal in treating excessive daytime sleepiness |
| 2022-06-06 | Harmony Biosciences Management, Inc. | Results HARMONY BIOSCIENCES PRESENTS POST-HOC ANALYSIS OF WAKIX® (PITOLISANT) PIVOTAL DATA IN ADULTS WITH HIGH BURDEN OF NARCOLEPSY SYMPTOMS harmonybiosciences.com ↗
The analysis was presented as a poster at the 36th Annual Meeting of the Associated Professional Sleep Societies (APSS), known as "SLEEP 2022," held in Charlotte, NC June 4-8 . |
| 2021-12-13 | Harmony Biosciences Management, Inc. | Results Harmony Biosciences Publishes Pitolisant Efficacy Data In CNS Drugs harmonybiosciences.com ↗ |
| 2021-12-01 | Harmony Biosciences Management, Inc. | Regulatory Harmony Biosciences Announces Plans To Initiate Phase 3 Clinical Trial For Treatment Of Idiopathic Hypersomnia harmonybiosciences.com ↗
IND for pitolisant for treatment of idiopathic hypersomnia accepted |
| 2021-06-10 | Harmony Biosciences Management, Inc. | Results Harmony Biosciences Presents New Analysis Of Clinical Impact For WAKIX® (pitolisant) At The SLEEP 2021 Annual Meeting harmonybiosciences.com ↗
This poster presents results from a new analysis that evaluated the clinical impact of pitolisant in adults with narcolepsy using NNT and effect size as measures of treatment response based on data from the HARMONY 1 and HARMONY CTP randomized, controlled clinical trials. |
All press releases naming this drug 35 releases
Evidence & citations 14 cited values
Every value below carries the sentence it was read from. 35 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Pitolisant | “Long Term Open Label Study in Narcolepsy With BF2.649 (Pitolisant)” NCT01399606 ↗ |
| Known as | BF-2649 HYDROCHLORIDE | ChEMBL registry synonym — accepted as the source's own label CHEMBL462605 ↗ |
| Known as | BF2.649 | “BF 2.649, a new molecule, reduces significantly the diurnal sleepiness and demonstrated its anti-cataplexy effect in pre-clinical and clinical studies.” NCT01067235 ↗16“A Trial Evaluating Pitolisant (BF2.649) in Alcohol Use Disorder Treatment” NCT02800083 ↗ “Pitolisant (BF2.649)” NCT02978651 ↗ “Pitolisant (BF2.649)” NCT02739568 ↗ |
| Known as | Ciproxidine | ChEMBL registry synonym — accepted as the source's own label CHEMBL462605 ↗ |
| Known as | HBS-101 | ChEMBL registry synonym — accepted as the source's own label CHEMBL462605 ↗ |
| Known as | Ozawade | ChEMBL registry synonym — accepted as the source's own label CHEMBL462605 ↗ |
| Known as | PITOLISANT HYDROCHLORIDE | ChEMBL registry synonym — accepted as the source's own label CHEMBL462605 ↗ |
| Known as | Tiprolisant | ChEMBL registry synonym — accepted as the source's own label CHEMBL462605 ↗ |
| Known as | Wakix | “Pitolisant 17.8 MG [Wakix]” NCT05849675 ↗1“Pitolisant (Wakix)” NCT05581576 ↗ |
| Known as | Wakix, europe | ChEMBL registry synonym — accepted as the source's own label CHEMBL462605 ↗ |
| Action | Inhibit | “inverse agonist at the histamine H3 receptor” NCT00642928 ↗ |
| Modality | Small molecule | — CHEMBL462605 ↗ |
| Route | Oral | “orally active” NCT00642928 ↗ |
| Target | HRH3 | “Histamine H3 receptor inverse agonist” CHEMBL462605 ↗ |