tezacaftor Indications/Contra | FAERs-F | FAERs-M | Orange Bk | BioActivity |

Stem definitionDrug idCAS RN
cystic fibrosis transmembrane regulator (CFTR) protein modulators 5276 1152311-62-0

Description:

MoleculeDescription

Molfile Inchi Smiles

Synonyms:

  • tezacaftor
  • VX-661
Tezacaftor facilitates the cellular processing and trafficking of normal and select mutant forms of CFTR (including F508del-CFTR) to increase the amount of mature CFTR protein delivered to the cell surface.
  • Molecular weight: 520.51
  • Formula: C26H27F3N2O6
  • CLOGP: 3.68
  • LIPINSKI: 1
  • HAC: 8
  • HDO: 4
  • TPSA: 113.18
  • ALOGS: -4.62
  • ROTB: 8

  • Status: ONP

  • Legend:
    OFP - off patent
    OFM - off market
    ONP - on patent

Drug dosage:

None

ADMET properties:

None

Approvals:

DateAgencyCompanyOrphan
Aug. 21, 2020 EMA Vertex Pharmaceuticals (Ireland) Limited
Feb. 12, 2018 FDA VERTEX PHARMS

FDA Adverse Event Reporting System (Female)

MedDRA adverse event termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients not taking drug not having adverse event
Infective pulmonary exacerbation of cystic fibrosis 3114.95 39.08 605 5295 10202 46669960
Hospitalisation 457.94 39.08 201 5699 63184 46616978
Cystic fibrosis 427.42 39.08 98 5802 3590 46676572
Cystic fibrosis respiratory infection suppression 260.53 39.08 46 5854 421 46679741
Infection 218.46 39.08 160 5740 133432 46546730
Haemoptysis 211.22 39.08 90 5810 26099 46654063
Productive cough 190.24 39.08 103 5797 50612 46629550
Pulmonary function test decreased 187.36 39.08 51 5849 3769 46676393
Headache 151.34 39.08 238 5662 478114 46202048
Cough 138.14 39.08 157 5743 230092 46450070
Abdominal pain upper 102.40 39.08 108 5792 145197 46534965
Influenza 86.43 39.08 79 5821 89191 46590971
Sputum increased 79.39 39.08 22 5878 1734 46678428
Distal intestinal obstruction syndrome 70.58 39.08 15 5885 382 46679780
Pneumonia 56.20 39.08 137 5763 376183 46303979
Forced expiratory volume decreased 52.82 39.08 21 5879 5083 46675079
Paranasal sinus hypersecretion 50.62 39.08 18 5882 3206 46676956
Emergency care 49.59 39.08 13 5887 829 46679333
Drug ineffective 44.96 39.08 15 5885 677823 46002339
Respiratory tract congestion 42.45 39.08 24 5876 12740 46667422

FDA Adverse Event Reporting System (Male)

MedDRA adverse event termLikelihood ratioLikelihood ratio thresholdPatients taking drug having adverse eventPatients taking drug not having adverse eventPatients not taking drug having adverse eventPatients not taking drug not having adverse event
Infective pulmonary exacerbation of cystic fibrosis 2013.94 51.18 409 3629 8163 29940277
Hospitalisation 335.54 51.18 148 3890 44171 29904269
Productive cough 239.14 51.18 104 3934 29849 29918591
Cystic fibrosis respiratory infection suppression 232.18 51.18 41 3997 350 29948090
Cystic fibrosis 195.16 51.18 48 3990 2226 29946214
Pulmonary function test decreased 151.71 51.18 45 3993 4250 29944190
Headache 146.04 51.18 149 3889 182157 29766283
Infection 141.23 51.18 102 3936 77972 29870468
Cough 135.21 51.18 121 3917 125521 29822919
Abdominal pain upper 129.97 51.18 89 3949 62462 29885978
Influenza 100.23 51.18 66 3972 43332 29905108
Lung transplant 94.25 51.18 26 4012 1890 29946550
Sputum increased 81.28 51.18 23 4015 1834 29946606
Haemoptysis 75.94 51.18 49 3989 31029 29917411
Nasopharyngitis 59.70 51.18 55 3983 58794 29889646
Testicular pain 56.06 51.18 19 4019 2753 29945687
Liver function test increased 55.32 51.18 28 4010 11188 29937252

Pharmacologic Action:

SourceCodeDescription
ATC R07AX31 RESPIRATORY SYSTEM
OTHER RESPIRATORY SYSTEM PRODUCTS
OTHER RESPIRATORY SYSTEM PRODUCTS
Other respiratory system products
ATC R07AX32 RESPIRATORY SYSTEM
OTHER RESPIRATORY SYSTEM PRODUCTS
OTHER RESPIRATORY SYSTEM PRODUCTS
Other respiratory system products

Drug Use (View source of the data)

DiseaseRelationSNOMED_IDDOID
Cystic fibrosis of the lung indication 86555001

Acid dissociation constants calculated using MoKa v3.0.0

Dissociation levelDissociation constantType (acidic/basic)
pKa1 12.64 acidic
pKa2 13.25 acidic
pKa3 13.99 acidic

Orange Book patent data (new drug applications)

None

Orange Book exclusivity data (new drug applications)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRouteExclusivity dateDescription
150MG,N/A;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET, TABLET ORAL June 21, 2022 NEW PATIENT POPULATION
75MG,N/A;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET, TABLET ORAL June 21, 2022 NEW PATIENT POPULATION
150MG,N/A;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET, TABLET ORAL Feb. 12, 2023 NEW CHEMICAL ENTITY
75MG,N/A;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET, TABLET ORAL Feb. 12, 2023 NEW CHEMICAL ENTITY
150MG,N/A;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET, TABLET ORAL Feb. 12, 2025 TREATMENT OF PATIENTS WITH CYSTIC FIBROSIS AGED 12 YEARS AND OLDER WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR WHO HAVE AT LEAST ONE MUTATION IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR GENE RESPONSIVE TO TEZACAFTOR/IVACAFTOR
75MG,N/A;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET, TABLET ORAL Feb. 12, 2025 TREATMENT OF PATIENTS WITH CYSTIC FIBROSIS AGED 12 YEARS AND OLDER WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR WHO HAVE AT LEAST ONE MUTATION IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR GENE RESPONSIVE TO TEZACAFTOR/IVACAFTOR
150MG,N/A;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET, TABLET ORAL June 21, 2026 TX OF PTS W/ CYSTIC FIBROSIS (CF) AGE 6 TO <12 YRS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR W/ AT LEAST 1 MUTATION IN CF TRANSMEMBRANE CONDUCTANCE REGULATORY GENE RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE
75MG,N/A;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET, TABLET ORAL June 21, 2026 TX OF PTS W/ CYSTIC FIBROSIS (CF) AGE 6 TO <12 YRS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR W/ AT LEAST 1 MUTATION IN CF TRANSMEMBRANE CONDUCTANCE REGULATORY GENE RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE
150MG,N/A;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET, TABLET ORAL Dec. 21, 2027 FOR TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 6 YEARS AND OLDER WHO HAVE AT LEAST ONE OF THE ADDITIONULLL MUTATIONS IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE THAT HAVE BEEN IDENTIFIED AS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND IDENTIFIED IN THE APPROVAL ON DECEMBER 21, 2020
75MG,N/A;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET, TABLET ORAL Dec. 21, 2027 FOR TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 6 YEARS AND OLDER WHO HAVE AT LEAST ONE OF THE ADDITIONULLL MUTATIONS IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE THAT HAVE BEEN IDENTIFIED AS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND IDENTIFIED IN THE APPROVAL ON DECEMBER 21, 2020

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
4037327 VANDF
C4519194 UMLSCUI
CHEMBL3544914 ChEMBL_ID
D11041 KEGG_DRUG
C000625213 MESH_SUPPLEMENTAL_RECORD_UI
10104 INN_ID
DB11712 DRUGBANK_ID
8RW88Y506K UNII
46199646 PUBCHEM_CID
10199 IUPHAR_LIGAND_ID
1999382 RXNORM
33763 MMSL
d08742 MMSL
017465 NDDF
771596001 SNOMEDCT_US

Pharmaceutical products:

None