Tezacaftor

RESPIRATORY SYSTEM

Key facts

Tezacaftor is an onp drug catalogued in DrugCentral. It was first approved by the FDA in 2018. DrugCentral lists 1 molecular target, including CFTR. Recorded indications include Cystic fibrosis of the lung.

• Approval status: onp (first approved 2018 by the FDA).
• Molecular targets: CFTR (1 listed in DrugCentral).
• Indications: Cystic fibrosis of the lung.
• ATC code: R07AX31 (Other respiratory system products).
• Molecular formula: C26H27F3N2O6; molecular weight 520.50 g/mol.

Indications & contraindications

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

Indications (1)

• Cystic fibrosis of the lung

Mechanism & targets

Molecular targets (1)

Target	Gene	Action	Activity
Cystic fibrosis transmembrane conductance regulator	CFTR	other	EC50 6.57

Regulatory approvals (2)

Date	Agency	Marketed as	Applicant	Orphan
2020-08-21	EMA	—	Vertex Pharmaceuticals (Ireland) Limited	Yes
2018-02-12	FDA	—	VERTEX PHARMS	Yes

ATC classification

ATC code	Therapeutic hierarchy
R07AX31	RESPIRATORY SYSTEM › OTHER RESPIRATORY SYSTEM PRODUCTS › OTHER RESPIRATORY SYSTEM PRODUCTS › Other respiratory system products
R07AX32	RESPIRATORY SYSTEM › OTHER RESPIRATORY SYSTEM PRODUCTS › OTHER RESPIRATORY SYSTEM PRODUCTS › Other respiratory system products
R07AX33	RESPIRATORY SYSTEM › OTHER RESPIRATORY SYSTEM PRODUCTS › OTHER RESPIRATORY SYSTEM PRODUCTS › Other respiratory system products

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
Pelvic kidney	85.0

Chemical structure

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

• Molecular formula: C₂₆H₂₇F₃N₂O₆
• Molecular weight: 520.50 g/mol
• CAS number: 1152311-62-0
• InChIKey: MJUVRTYWUMPBTR-MRXNPFEDSA-N
• PubChem CID: 46199646

CC(C)(CO)C1=CC2=C(C=C(F)C(NC(=O)C3(CC3)C3=CC=C4OC(F)(F)OC4=C3)=C2)N1C[C@@H](O)CO

Frequently asked questions

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

What is Tezacaftor used for?

DrugCentral lists the following indications for Tezacaftor: Cystic fibrosis of the lung.

How does Tezacaftor work?

Its molecular targets in DrugCentral include CFTR.

When was Tezacaftor first approved?

Tezacaftor was first approved by the FDA in 2018.

What is the chemical formula of Tezacaftor?

Tezacaftor has the molecular formula C26H27F3N2O6 and a molecular weight of 520.50 g/mol.

Sources

• DrugCentral drug card 5276 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL3544914 — compound record and bioactivity
• PubChem CID 46199646 — chemical identifiers
• DrugBank DB11712 — drug record
• FDA UNII 8RW88Y506K — substance identity
• DailyMed: FDA labels for Tezacaftor — current prescribing information (label search)
• UniProt P13569 (CFTR) — 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.