vanzacaftor 🐶 Veterinary Use | Indications/Contra | FAERs-F | FAERs-M | Orange Bk | PK/Tox | Related Drugs | BioActivity |

Stem definitionDrug idCAS RN
cystic fibrosis transmembrane regulator (CFTR) protein modulators, correctors, and amplifiers 5847 None

Description:

MoleculeDescription

Molfile Inchi Smiles

Synonyms:

  • vanzacaftor
  • vanzacaftor calcium dihydrate
ALYFTREK is a combination of vanzacaftor, tezacaftor, and deutivacaftor that was approved for the treatment of cystic fibrosis. Vanzacaftor and tezacaftor bind to different sites on the CFTR protein and have an additive effect in facilitating the cellular processing and trafficking of select mutant forms of CFTR (including F508del-CFTR) to increase the amount of CFTR protein delivered to the cell surface compared to either molecule alone. Deutivacaftor potentiates the channel open probability (or gating) of the CFTR protein at the cell surface.
  • Molecular weight: 617.77
  • Formula: C32H39N7O4S
  • CLOGP: 4.55
  • LIPINSKI: 3
  • HAC: 11
  • HDO: 2
  • TPSA: 131.34
  • ALOGS:
  • ROTB: 5

Drug dosage:

None

ADMET properties:

None

Predicted pharmacokinetic/toxicological properties (PK-AZ):

None

Approvals:

DateAgencyCompanyOrphan
June 30, 2025 EMA Vertex Pharmaceuticals (Ireland) Limited
Dec. 20, 2024 FDA Vertex Pharmaceuticals

FDA Adverse Event Reporting System (Female)

None

FDA Adverse Event Reporting System (Male)

None

FDA Adverse Event Reporting System (Geriatric)

None

FDA Adverse Event Reporting System (Pediatric)

None

Pharmacologic Action:

SourceCodeDescription
ATC R07AX33 RESPIRATORY SYSTEM
OTHER RESPIRATORY SYSTEM PRODUCTS
OTHER RESPIRATORY SYSTEM PRODUCTS
Other respiratory system products

Related Drugs by ATC class:

R07AX Other respiratory system products 6 drugs

Drug Use | Suggest Off label Use Form| |View source of the data|

DiseaseRelationSNOMED_IDDOID
Cystic fibrosis indication 190905008 DOID:1485




🐶 Veterinary Drug Use

None

🐶 Veterinary products

None

Acid dissociation constants calculated using MoKa v3.0.0

None

Orange Book patent data (new drug applications)

None

Orange Book exclusivity data (new drug applications)

None

Bioactivity Summary:

TargetClassPharosUniProtActionTypeActivity value
(-log[M])
Mechanism
action
Bioact sourceMoA source
Cystic fibrosis transmembrane conductance regulator Ion channel PHARMACOLOGICAL CHAPERONE UNKNOWN DRUG LABEL

External reference:

IDSource
2374124-49-7 SECONDARY_CAS_RN
CHEMBL5314934 ChEMBL_ID
C000731979 MESH_SUPPLEMENTAL_RECORD_UI
4043970 VANDF
C5707868 UMLSCUI
CHEBI:759626 CHEBI
11971 INN_ID
2607828-37-3 SECONDARY_CAS_RN
139399801 PUBCHEM_CID
13694 IUPHAR_LIGAND_ID
COM1POP492 UNII
2701431 RXNORM
45314111000001106 SNOMEDCT_US
45314211000001100 SNOMEDCT_US
D12718 KEGG_DRUG
43614 MMSL
d10240 MMSL
019956 NDDF

Pharmaceutical products:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
ALYFTREK HUMAN PRESCRIPTION DRUG LABEL 3 51167-121 TABLET, FILM COATED 10 mg ORAL NDA 33 sections
ALYFTREK HUMAN PRESCRIPTION DRUG LABEL 3 51167-135 TABLET, FILM COATED 4 mg ORAL NDA 33 sections