Danicopan

Complement Factor D Inhibitor — ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS

Key facts

Danicopan is an onp drug in the Complement Factor D Inhibitor class catalogued in DrugCentral. It was first approved by the PMDA in 2024. Its mechanism of action is classified as Breast Cancer Resistance Protein Inhibitors, Complement Factor D Inhibitors and P-Glycoprotein Inhibitors. DrugCentral lists 1 molecular target, including CFD. Recorded indications include Paroxysmal nocturnal hemoglobinuria.

• Approval status: onp (first approved 2024 by the PMDA).
• Drug class: Complement Factor D Inhibitor.
• Mechanism of action: Breast Cancer Resistance Protein Inhibitors; Complement Factor D Inhibitors; P-Glycoprotein Inhibitors.
• Molecular targets: CFD (1 listed in DrugCentral).
• Indications: Paroxysmal nocturnal hemoglobinuria.
• ATC code: L04AJ09 (Complement inhibitors).
• Molecular formula: C26H23BrFN7O3; molecular weight 580.42 g/mol.

Indications & contraindications

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

Indications (1)

• Paroxysmal nocturnal hemoglobinuria

Mechanism & targets

• Mechanism of action (MoA): Breast Cancer Resistance Protein Inhibitors; Complement Factor D Inhibitors; P-Glycoprotein Inhibitors
• Established pharmacologic class: Complement Factor D Inhibitor

Molecular targets (1)

Target	Gene	Action	Activity
Complement factor D	CFD	other	Kd 9.30

Regulatory approvals (3)

Date	Agency	Marketed as	Applicant	Orphan
2024-04-19	EMA	—	Alexion Europe SAS	Yes
2024-03-30	FDA	—	Alexion Pharmaceuticals Inc.	Yes
2024-01-08	PMDA	—	Alexion Pharma GK	Yes

ATC classification

ATC code	Therapeutic hierarchy
L04AJ09	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
Haemolysis	111.3
Extravascular haemolysis	110.7
Breakthrough haemolysis	92.2

Chemical structure

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

• Molecular formula: C₂₆H₂₃BrFN₇O₃
• Molecular weight: 580.42 g/mol
• CAS number: —
• InChIKey: PIBARDGJJAGJAJ-NQIIRXRSSA-N
• PubChem CID: 118323590

CC1=NC=C(C=N1)C2=CC3=C(C=C2)N(N=C3C(=O)C)CC(=O)N4C[C@@H](C[C@H]4C(=O)NC5=NC(=CC=C5)Br)F

Frequently asked questions

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

What is Danicopan used for?

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

How does Danicopan work?

Danicopan is classified as Breast Cancer Resistance Protein Inhibitors, Complement Factor D Inhibitors and P-Glycoprotein Inhibitors; its molecular targets in DrugCentral include CFD.

When was Danicopan first approved?

Danicopan was first approved by the PMDA in 2024.

What drug class does Danicopan belong to?

Danicopan is in the Complement Factor D Inhibitor class.

What is the chemical formula of Danicopan?

Danicopan has the molecular formula C26H23BrFN7O3 and a molecular weight of 580.42 g/mol.

Sources

• DrugCentral drug card 5816 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL4250860 — compound record and bioactivity
• PubChem CID 118323590 — chemical identifiers
• DrugBank DB15401 — drug record
• FDA UNII JM8C1SFX0U — substance identity
• DailyMed: FDA labels for Danicopan — current prescribing information (label search)
• UniProt P00746 (CFD) — 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.