Pitolisant

Histamine-3 Receptor Antagonist/Inverse Agonist — NERVOUS SYSTEM

Key facts

Pitolisant is an onp drug in the Histamine-3 Receptor Antagonist/Inverse Agonist class catalogued in DrugCentral. It was first approved by the EMA in 2016. Its mechanism of action is classified as Cytochrome P450 3A4 Inducers, Histamine H3 Receptor Antagonists and Histamine H3 Receptor Inverse Agonists. DrugCentral lists 16 molecular targets, including HRH3, HRH3 and SIGMAR1. Recorded indications include Narcolepsy.

• Approval status: onp (first approved 2016 by the EMA).
• Drug class: Histamine-3 Receptor Antagonist/Inverse Agonist.
• Mechanism of action: Cytochrome P450 3A4 Inducers; Histamine H3 Receptor Antagonists; Histamine H3 Receptor Inverse Agonists.
• Molecular targets: HRH3, HRH3, SIGMAR1 and SIGMAR1 (16 listed in DrugCentral).
• Indications: Narcolepsy.
• ATC code: N07XX11 (Other nervous system drugs).
• Molecular formula: C17H26ClNO; molecular weight 295.85 g/mol.

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (1)

• Narcolepsy

Mechanism & targets

• Mechanism of action (MoA): Cytochrome P450 3A4 Inducers; Histamine H3 Receptor Antagonists; Histamine H3 Receptor Inverse Agonists
• Established pharmacologic class: Histamine-3 Receptor Antagonist/Inverse Agonist

Molecular targets (16)

Target	Gene	Action	Activity
Histamine H3 receptor	HRH3	unknown	Ki 9.80
Histamine H3 receptor	HRH3	inverse agonist	Ki 9.80
Sigma non-opioid intracellular receptor 1	SIGMAR1	unknown	Ki 9.30
Sigma non-opioid intracellular receptor 1	SIGMAR1	unknown	Ki 9.04
Sigma intracellular receptor 2	Tmem97	unknown	Ki 8.19
Transmembrane protein 97	TMEM97	unknown	Ki 8.15
Histamine H3 receptor	Hrh3	unknown	Ki 7.85
Histamine H3 receptor	Hrh3	unknown	Ki 7.77
Muscarinic acetylcholine receptor M4	CHRM4	unknown	Ki 6.05
5-hydroxytryptamine receptor 7	HTR7	unknown	Ki 5.96
Histamine H1 receptor	HRH1	unknown	Ki 5.94
Potassium voltage-gated channel subfamily H member 2	KCNH2	unknown	IC50 5.77
Sodium-dependent noradrenaline transporter	SLC6A2	unknown	Ki 5.72
5-hydroxytryptamine receptor 2C	HTR2C	unknown	Ki 5.70
Cholinesterase	BCHE	unknown	IC50 5.08
Histamine H2 receptor	HRH2	antagonist	Ki 4.96

Regulatory approvals (2)

Date	Agency	Marketed as	Applicant	Orphan
2019-08-14	FDA	—	HARMONY
2016-03-31	EMA	—	Bioprojet Pharma	Yes

ATC classification

ATC code	Therapeutic hierarchy
N07XX11	NERVOUS SYSTEM › OTHER NERVOUS SYSTEM DRUGS › OTHER NERVOUS SYSTEM DRUGS › Other nervous system drugs

Pharmacogenomics

Drug-label and guideline annotations from PharmGKB and CPIC.

• EMA label: CYP2D6 · Actionable PGx
• FDA label: CYP2D6 · Actionable PGx
• HCSC label: CYP2D6 · Actionable PGx

Reported adverse-event signals

Strongest FDA FAERS disproportionality signals (log-likelihood ratio). A signal is a statistical association in spontaneous reports, not proof that the drug causes the event.

MedDRA term	LLR
Product administration interrupted	456.7
Surgery	120.7
Anxiety	119.6
Splenic infarction	87.6
Suicidal ideation	81.3
Intentional dose omission	76.0
Pre-existing condition improved	66.1
Cataplexy	60.1
Somnambulism	56.2
Depression	55.9
Sleep apnoea syndrome	50.6
Enuresis	49.2

Chemical structure

• Molecular formula: C₁₇H₂₆ClNO
• Molecular weight: 295.85 g/mol
• CAS number: —
• InChIKey: NNACHAUCXXVJSP-UHFFFAOYSA-N
• PubChem CID: 9948102

ClC1=CC=C(CCCOCCCN2CCCCC2)C=C1

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Pitolisant used for?

DrugCentral lists the following indications for Pitolisant: Narcolepsy.

How does Pitolisant work?

Pitolisant is classified as Cytochrome P450 3A4 Inducers, Histamine H3 Receptor Antagonists and Histamine H3 Receptor Inverse Agonists; its molecular targets in DrugCentral include HRH3, HRH3 (inverse agonist), SIGMAR1 and SIGMAR1.

When was Pitolisant first approved?

Pitolisant was first approved by the EMA in 2016.

What drug class does Pitolisant belong to?

Pitolisant is in the Histamine-3 Receptor Antagonist/Inverse Agonist class.

What is the chemical formula of Pitolisant?

Pitolisant has the molecular formula C17H26ClNO and a molecular weight of 295.85 g/mol.

Sources

• DrugCentral drug card 5048 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL4164059 — compound record and bioactivity
• PubChem CID 9948102 — chemical identifiers
• DrugBank DB11642 — drug record
• FDA UNII 4BC83L4PIY — substance identity
• DailyMed: FDA labels for Pitolisant — current prescribing information (label search)
• UniProt Q9Y5N1 (HRH3) — target protein
• UniProt Q99720 (SIGMAR1) — target protein
• UniProt Q5BJF2 (TMEM97) — target protein
• UniProt P08173 (CHRM4) — target protein
• UniProt P34969 (HTR7) — target protein
• UniProt P35367 (HRH1) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral
• PharmGKB / CPIC — pharmacogenomic label annotations

How this page is built: methodology · editorial policy & corrections.