Vanzacaftor

RESPIRATORY SYSTEM

Key facts

Vanzacaftor is a drug catalogued in DrugCentral. It was first approved by the FDA in 2024. DrugCentral lists 1 molecular target, including CFTR. Recorded indications include Cystic fibrosis.

• Molecular targets: CFTR (1 listed in DrugCentral).
• Indications: Cystic fibrosis.
• ATC code: R07AX33 (Other respiratory system products).
• Molecular formula: C32H39N7O4S; molecular weight 617.78 g/mol.

Indications & contraindications

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

Indications (1)

• Cystic fibrosis

Mechanism & targets

Molecular targets (1)

Target	Gene	Action	Activity
Cystic fibrosis transmembrane conductance regulator	CFTR	other	—

Regulatory approvals (2)

Date	Agency	Marketed as	Applicant	Orphan
2025-06-30	EMA	—	Vertex Pharmaceuticals (Ireland) Limited	Yes
2024-12-20	FDA	—	Vertex Pharmaceuticals	Yes

ATC classification

ATC code	Therapeutic hierarchy
R07AX33	RESPIRATORY SYSTEM › OTHER RESPIRATORY SYSTEM PRODUCTS › OTHER RESPIRATORY SYSTEM PRODUCTS › Other respiratory system products

Chemical structure

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

• Molecular formula: C₃₂H₃₉N₇O₄S
• Molecular weight: 617.78 g/mol
• CAS number: —
• InChIKey: VCSUIBJKYCVWNF-NRFANRHFSA-N
• PubChem CID: 139399801

CC1(C[C@@H]2CCCNC3=NC(=CC=C3)S(=O)(=O)NC(=O)C4=C(N1C2)N=C(C=C4)N5C=CC(=N5)OCCC6C7(C68CC8)CC7)C

Frequently asked questions

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

What is Vanzacaftor used for?

DrugCentral lists the following indications for Vanzacaftor: Cystic fibrosis.

How does Vanzacaftor work?

Its molecular targets in DrugCentral include CFTR.

When was Vanzacaftor first approved?

Vanzacaftor was first approved by the FDA in 2024.

What is the chemical formula of Vanzacaftor?

Vanzacaftor has the molecular formula C32H39N7O4S and a molecular weight of 617.78 g/mol.

Sources

• DrugCentral drug card 5847 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL5314934 — compound record and bioactivity
• PubChem CID 139399801 — chemical identifiers
• FDA UNII COM1POP492 — substance identity
• DailyMed: FDA labels for Vanzacaftor — current prescribing information (label search)
• UniProt P13569 (CFTR) — target protein

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