GtoPdb Ligand ID: 8924

Synonyms: BF2.649 | tiprolisant [USAN] | Wakix®
pitolisant is an approved drug (EMA (2016), FDA (2019))
Compound class: Synthetic organic
Comment: Pitolisant is a selective antagonist/inverse agonist of the histamine H3 receptor [7-8,11].
2D Structure
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Physico-chemical Properties
Hydrogen bond acceptors 2
Hydrogen bond donors 0
Rotatable bonds 8
Topological polar surface area 12.47
Molecular weight 295.17
XLogP 3.94
No. Lipinski's rules broken 0
Canonical SMILES Clc1ccc(cc1)CCCOCCCN1CCCCC1
Isomeric SMILES Clc1ccc(cc1)CCCOCCCN1CCCCC1
InChI InChI=1S/C17H26ClNO/c18-17-9-7-16(8-10-17)6-4-14-20-15-5-13-19-11-2-1-3-12-19/h7-10H,1-6,11-15H2
No information available.
Summary of Clinical Use
Pitolisant was initially granted orphan designation as a treatment for excessive daytime sleepiness and cataplexy in patients with narcolspey in the US and across Europe. In March 2016, pitolisant received EMA marketing authorisation for the treatment of narcolepsy [2]. Phase 3 trial results were reported in 2017 [10]. It was 2019 before the FDA approved this drug for the same indication as covered by the EMA marketing authorisation.
Mechanism Of Action and Pharmacodynamic Effects
Preclinical pharmacology of this compound is reported in [4-5], and its clinical potential is discussed in [1,3,9].
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