Drugs / Pitolisant

Regulatory milestones approvals, filings & regulatory actions · 4 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
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 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 15 trials
Phase 1 NCT04596267 Sep 2021 → Feb 2023 alcohol-related disorders Boston Medical Center Terminated No outcome recorded
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Phase 2 NCT02800083 Oct 2016 → Dec 2019 alcohol abuse Bioprojet Withdrawn No outcome recorded
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 318 trials
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 NCT05156047 May 2022 → Sep 2023 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 No outcome recorded
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 No outcome recorded
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 No outcome recorded
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
Also used as a comparator or background therapy in 2 trials
PhaseRegistry idDatesIndicationSponsorStatusOutcome
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

Press releases naming this drug 1 release

DateIssuerRelease
2025-10-15 Pfizer's Upjohn has merged with Mylan to form Viatris Inc. Viatris Completes Acquisition of Aculys Pharma Including Exclusive Rights to Pitolisant in Japan and to Spydia® in Japan and Certain Other Markets in the Asia-Pacific Region viatris.com

Evidence & citations 14 cited values

Every value below carries the sentence it was read from. 34 sources stand behind the page.

FieldValueCited text
Known as Pitolisant “Long Term Open Label Study in Narcolepsy With BF2.649 (Pitolisant)” NCT01399606
20

NCT02800083

NCT01800045

NCT05223166

NCT04596267

NCT05581576

NCT05953389

NCT04886518

NCT04026750

NCT02978651

NCT05458128

NCT07219485

NCT03152123

NCT05156047

NCT04257929

NCT03433131

NCT05849675

NCT02929342

NCT06366464

NCT02611687

NCT02739568

Known as BF 2.649 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)” NCT02739568

“Pitolisant (BF2.649)” NCT02978651

NCT01789398

NCT00642928

NCT01066442

NCT01036139

NCT01619033

NCT01067222

NCT01067235

NCT01071876

NCT00690274

NCT01620554

NCT01072968

NCT01638403

NCT01399606

Known as BF-2649 HYDROCHLORIDE ChEMBL registry synonym — accepted as the source's own label CHEMBL462605
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