Iptacopan

Complement Factor B Inhibitor — ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS

Key facts

Iptacopan is an onp drug in the Complement Factor B Inhibitor class catalogued in DrugCentral. It was first approved by the FDA in 2023. Its mechanism of action is classified as Complement Factor B Inhibitors. DrugCentral lists 2 molecular targets, including CFB and ADRA1A. Recorded indications include Paroxysmal nocturnal hemoglobinuria.

• Approval status: onp (first approved 2023 by the FDA).
• Drug class: Complement Factor B Inhibitor.
• Mechanism of action: Complement Factor B Inhibitors.
• Molecular targets: CFB and ADRA1A (2 listed in DrugCentral).
• Indications: Paroxysmal nocturnal hemoglobinuria.
• ATC code: L04AJ08 (Complement inhibitors).
• Molecular formula: C25H30N2O4; molecular weight 422.52 g/mol.

Indications & contraindications

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

Indications (1)

• Paroxysmal nocturnal hemoglobinuria

Contraindications (1)

• Unresolved serious infection caused by encapsulated bacteria

Mechanism & targets

• Mechanism of action (MoA): Complement Factor B Inhibitors
• Established pharmacologic class: Complement Factor B Inhibitor

Molecular targets (2)

Target	Gene	Action	Activity
Complement factor B	CFB	inhibitor	IC50 8.02
Alpha-1A adrenergic receptor	ADRA1A	unknown	IC50 7.92

Regulatory approvals (1)

Date	Agency	Marketed as	Applicant	Orphan
2023-12-05	FDA	—	NOVARTIS PHARMS CORP	Yes

ATC classification

ATC code	Therapeutic hierarchy
L04AJ08	ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS › IMMUNOSUPPRESSANTS › IMMUNOSUPPRESSANTS › Complement inhibitors

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
Breakthrough haemolysis	206.0
Haemolysis	49.3

Chemical structure

Full physicochemical, ADMET and 3D-structure analysis of Iptacopan is on its molecule page.

• Molecular formula: C₂₅H₃₀N₂O₄
• Molecular weight: 422.52 g/mol
• CAS number: —
• InChIKey: RENRQMCACQEWFC-UGKGYDQZSA-N
• PubChem CID: 90467622

CCO[C@H]1CCN(CC2=C(OC)C=C(C)C3=C2C=CN3)[C@@H](C1)C1=CC=C(C=C1)C(O)=O

Frequently asked questions

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

What is Iptacopan used for?

DrugCentral lists the following indications for Iptacopan: Paroxysmal nocturnal hemoglobinuria.

How does Iptacopan work?

Iptacopan is classified as Complement Factor B Inhibitors; its molecular targets in DrugCentral include CFB (inhibitor) and ADRA1A.

When was Iptacopan first approved?

Iptacopan was first approved by the FDA in 2023.

What drug class does Iptacopan belong to?

Iptacopan is in the Complement Factor B Inhibitor class.

What is the chemical formula of Iptacopan?

Iptacopan has the molecular formula C25H30N2O4 and a molecular weight of 422.52 g/mol.

Sources

• DrugCentral drug card 5754 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL4594448 — compound record and bioactivity
• PubChem CID 90467622 — chemical identifiers
• DrugBank DB16200 — drug record
• FDA UNII 8E05T07Z6W — substance identity
• DailyMed: FDA labels for Iptacopan — current prescribing information (label search)
• UniProt P00751 (CFB) — target protein
• UniProt P35348 (ADRA1A) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral

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