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 3203.66 33.15 626 6300 10530 53331610
Hospitalisation 446.01 33.15 206 6720 70806 53271334
Cystic fibrosis 444.94 33.15 103 6823 3828 53338312
Cystic fibrosis respiratory infection suppression 270.68 33.15 48 6878 438 53341702
Haemoptysis 229.00 33.15 98 6828 27941 53314199
Pulmonary function test decreased 214.00 33.15 58 6868 4103 53338037
Productive cough 192.83 33.15 109 6817 56620 53285520
Infection 180.62 33.15 161 6765 172044 53170096
Cough 139.95 33.15 169 6757 256724 53085416
Headache 137.53 33.15 247 6679 536574 52805566
Abdominal pain upper 108.13 33.15 120 6806 166171 53175969
Sputum increased 100.16 33.15 28 6898 2218 53339922
Influenza 81.97 33.15 80 6846 95299 53246841
Distal intestinal obstruction syndrome 72.65 33.15 16 6910 470 53341670
Paranasal sinus hypersecretion 64.69 33.15 22 6904 3344 53338796
Drug ineffective 58.55 33.15 17 6909 817228 52524912
Pneumonia 56.86 33.15 147 6779 407022 52935118
Weight increased 54.27 33.15 96 6830 204471 53137669
Forced expiratory volume decreased 53.83 33.15 22 6904 5563 53336577
Sinus operation 52.73 33.15 16 6910 1689 53340451
Exposure during pregnancy 50.30 33.15 71 6855 124789 53217351
Acne 48.79 33.15 32 6894 21554 53320586
Emergency care 48.27 33.15 13 6913 895 53341245
Abortion spontaneous 45.50 33.15 42 6884 46593 53295547
Anxiety 43.01 33.15 85 6841 196619 53145521
Respiratory tract congestion 41.26 33.15 25 6901 14674 53327466
Acne cystic 39.92 33.15 10 6916 517 53341623
Increased appetite 37.74 33.15 20 6906 9102 53333038
Rhinovirus infection 36.48 33.15 15 6911 3850 53338290
Rash pruritic 35.71 33.15 38 6888 49972 53292168
Constipation 35.50 33.15 79 6847 198333 53143807
Rash 34.22 33.15 131 6795 446060 52896080

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 2035.34 47.88 417 4117 8362 32500630
Hospitalisation 335.08 47.88 154 4380 48817 32460175
Productive cough 235.16 47.88 108 4426 34008 32474984
Cystic fibrosis respiratory infection suppression 230.77 47.88 41 4493 350 32508642
Cystic fibrosis 201.65 47.88 50 4484 2316 32506676
Pulmonary function test decreased 147.86 47.88 46 4488 4929 32504063
Headache 143.01 47.88 155 4379 196042 32312950
Infection 137.18 47.88 105 4429 84610 32424382
Abdominal pain upper 128.10 47.88 92 4442 67246 32441746
Cough 123.13 47.88 122 4412 138695 32370297
Influenza 99.36 47.88 68 4466 45946 32463046
Lung transplant 97.09 47.88 27 4507 1953 32507039
Haemoptysis 78.19 47.88 52 4482 33473 32475519
Sputum increased 77.02 47.88 23 4511 2142 32506850
Testicular pain 70.12 47.88 23 4511 2913 32506079
Liver function test increased 65.75 47.88 33 4501 12527 32496465
Nasopharyngitis 56.17 47.88 56 4478 63731 32445261
Paranasal sinus hypersecretion 48.25 47.88 13 4521 834 32508158

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)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRoutePatent numberPatent expiration datePatent use
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 8629162 June 24, 2025 TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF PATIENTS WITH A MILD TO MODERATE CLINICAL PHENOTYPE OF CYSTIC FIBROSIS HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 8629162 June 24, 2025 TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF PATIENTS WITH A MILD TO MODERATE CLINICAL PHENOTYPE OF CYSTIC FIBROSIS HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 8354427 July 6, 2026 TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 6 YEARS AND OLDER WHO HAVE A R117H MUTATION IN THE CFTR GENE
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 8354427 July 6, 2026 TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 6 YEARS AND OLDER WHO HAVE A R117H MUTATION IN THE CFTR GENE
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 9670163 Dec. 28, 2026 TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 9931334 Dec. 28, 2026 TREATING CYSTIC FIBROSIS PATIENTS AGES 12 AND OLDER, WHO ARE HOMOZYGOUS FOR F508DEL OR HAVE AT LEAST 1 CFTR GENE MUTATION RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND A SOLID COMPOSITION COMPRISING AMORPHOUS (<30% CRYSTALLINE) IVACAFTOR
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 9931334 Dec. 28, 2026 TREATING CYSTIC FIBROSIS PATIENTS AGES 6 AND OLDER, WHO ARE HOMOZYGOUS FOR F508DEL OR HAVE AT LEAST 1 CFTR GENE MUTATION RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND A SOLID COMPOSITION COMPRISING AMORPHOUS (<30% CRYSTALLINE) IVACAFTOR
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 9670163 Dec. 28, 2026 TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 9931334 Dec. 28, 2026 TREATING CYSTIC FIBROSIS PATIENTS AGES 12 AND OLDER, WHO ARE HOMOZYGOUS FOR F508DEL OR HAVE AT LEAST 1 CFTR GENE MUTATION RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND A SOLID COMPOSITION COMPRISING AMORPHOUS (<30% CRYSTALLINE) IVACAFTOR
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 9931334 Dec. 28, 2026 TREATING CYSTIC FIBROSIS PATIENTS AGES 6 AND OLDER, WHO ARE HOMOZYGOUS FOR F508DEL OR HAVE AT LEAST 1 CFTR GENE MUTATION RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND A SOLID COMPOSITION COMPRISING AMORPHOUS (<30% CRYSTALLINE) IVACAFTOR
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 10022352 April 9, 2027 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 12 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HETEROZYGOUS FOR F508DEL AND A SECOND CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND IVACAFTOR
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 10022352 April 9, 2027 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HETEROZYGOUS FOR F508DEL AND A SECOND CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND IVACAFTOR
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 10239867 April 9, 2027 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 12 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH AN EFFECTIVE AMOUNT OF TEZACAFTOR AND IVACAFTOR
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 10239867 April 9, 2027 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH AN EFFECTIVE AMOUNT OF TEZACAFTOR AND IVACAFTOR
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 9974781 April 9, 2027 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 12 AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND IVACAFTOR
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 9974781 April 9, 2027 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND IVACAFTOR
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 10022352 April 9, 2027 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 12 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HETEROZYGOUS FOR F508DEL AND A SECOND CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND IVACAFTOR
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 10022352 April 9, 2027 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HETEROZYGOUS FOR F508DEL AND A SECOND CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND IVACAFTOR
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 10239867 April 9, 2027 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 12 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH AN EFFECTIVE AMOUNT OF TEZACAFTOR AND IVACAFTOR
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 10239867 April 9, 2027 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 YEARS AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH AN EFFECTIVE AMOUNT OF TEZACAFTOR AND IVACAFTOR
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 9974781 April 9, 2027 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 12 AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND IVACAFTOR
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 9974781 April 9, 2027 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGED 6 AND OLDER, WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVE AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH TEZACAFTOR AND IVACAFTOR
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 8598181 May 1, 2027 TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 8598181 May 1, 2027 TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 8324242 Aug. 5, 2027 TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 8324242 Aug. 5, 2027 TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 8415387 Nov. 12, 2027 TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 8415387 Nov. 12, 2027 TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HAVING AT LEAST ONE CFTR GENE MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR BASED ON IN VITRO DATA AND/OR CLINICAL EVIDENCE
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 10081621 March 25, 2031 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 12 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR WHO HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,081,621
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 10081621 March 25, 2031 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 6 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR WHO HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,081,621
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 10081621 March 25, 2031 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGES 12 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR ONE F508DEL MUTATION AND A CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,081,621
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 10081621 March 25, 2031 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGES 6 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR ONE F508DEL MUTATION AND A CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,081,621
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 10081621 March 25, 2031 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 12 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR WHO HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,081,621
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 10081621 March 25, 2031 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 6 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR WHO HAVE AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,081,621
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 10081621 March 25, 2031 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGES 12 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR ONE F508DEL MUTATION AND A CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,081,621
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 10081621 March 25, 2031 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGES 6 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR ONE F508DEL MUTATION AND A CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,081,621
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 10058546 July 15, 2033 TREATMENT OF CF IN PATIENTS 12 YEARS AND OLDER WHO HAVE A F508DEL OR G551D CFTR MUTATION AND A 2ND MUTATION SELECTED FROM R117H, A455E, 2789+5G->A, & 3849+10KBC->T, COMPRISING CONCURRENT COADMINISTRATION OF THE COMPOSITIONS OF CLAIM 1 OF US 10058546
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 10058546 July 15, 2033 TREATMENT OF CF IN PATIENTS 6 YEARS AND OLDER WHO HAVE A F508DEL OR G551D CFTR MUTATION AND A 2ND MUTATION SELECTED FROM R117H, A455E, 2789+5G->A, AND 3849+10KBC->T, COMPRISING CONCURRENT COADMINISTRATION OF THE COMPOSITIONS OF CLAIM 1 OF US 10058546
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 10058546 July 15, 2033 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS 12 YEARS AND OLDER, WITH A F508DEL OR G551D CFTR GENE MUTATION AND A A455E, 2789+5G->A, OR 3849+10KBC->T MUTATION, COMPRISING CONCURRENT COADMINISTRATION OF THE COMPOSITIONS OF CLAIM 1 OF U.S PATENT 10058546
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 10058546 July 15, 2033 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS 6 YEARS AND OLDER, WITH A F508DEL OR G551D CFTR GENE MUTATION AND A A455E, 2789+5G->, OR 3849+10KBC->T MUTATION, COMPRISING CONCURRENT COADMINISTRATION OF THE COMPOSITIONS OF CLAIM 1 OF U.S. PATENT 10058546
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 9012496 July 15, 2033 TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HETEROZYGOUS FOR THE F508DEL MUTATION AND A SECOND MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 10058546 July 15, 2033 TREATMENT OF CF IN PATIENTS 12 YEARS AND OLDER WHO HAVE A F508DEL OR G551D CFTR MUTATION AND A 2ND MUTATION SELECTED FROM R117H, A455E, 2789+5G->A, & 3849+10KBC->T, COMPRISING CONCURRENT COADMINISTRATION OF THE COMPOSITIONS OF CLAIM 1 OF US 10058546
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 10058546 July 15, 2033 TREATMENT OF CF IN PATIENTS 6 YEARS AND OLDER WHO HAVE A F508DEL OR G551D CFTR MUTATION AND A 2ND MUTATION SELECTED FROM R117H, A455E, 2789+5G->A, AND 3849+10KBC->T, COMPRISING CONCURRENT COADMINISTRATION OF THE COMPOSITIONS OF CLAIM 1 OF US 10058546
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 10058546 July 15, 2033 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS 12 YEARS AND OLDER, WITH A F508DEL OR G551D CFTR GENE MUTATION AND A A455E, 2789+5G->A, OR 3849+10KBC->T MUTATION, COMPRISING CONCURRENT COADMINISTRATION OF THE COMPOSITIONS OF CLAIM 1 OF U.S PATENT 10058546
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 10058546 July 15, 2033 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS 6 YEARS AND OLDER, WITH A F508DEL OR G551D CFTR GENE MUTATION AND A A455E, 2789+5G->, OR 3849+10KBC->T MUTATION, COMPRISING CONCURRENT COADMINISTRATION OF THE COMPOSITIONS OF CLAIM 1 OF U.S. PATENT 10058546
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 9012496 July 15, 2033 TEZACAFTOR AND IVACAFTOR FOR THE TREATMENT OF CYSTIC FIBROSIS IN PATIENTS WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION OR HETEROZYGOUS FOR THE F508DEL MUTATION AND A SECOND MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 10206877 April 14, 2035 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 12 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,206,877
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 10206877 April 14, 2035 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 6 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,206,877
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 10206877 April 14, 2035 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGES 12 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR ONE F508DEL MUTATION AND A CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,206,877
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL 10206877 April 14, 2035 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGES 6 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR ONE F508DEL MUTATION AND A CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,206,877
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 10206877 April 14, 2035 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 12 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,206,877
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 10206877 April 14, 2035 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 6 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR AT LEAST ONE CFTR MUTATION THAT IS RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,206,877
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 10206877 April 14, 2035 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGES 12 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR ONE F508DEL MUTATION AND A CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,206,877
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL 10206877 April 14, 2035 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGES 6 AND OLDER, WHO HAVE TWO COPIES OF THE F508DEL MUTATION OR ONE F508DEL MUTATION AND A CFTR MUTATION PREDICTED TO BE RESPONSIVE TO TEZACAFTOR/IVACAFTOR, WITH THE COMPOSITION OF CLAIM 1 OF US 10,206,877

Orange Book exclusivity data (new drug applications)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRouteExclusivity dateDescription
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL June 21, 2022 NEW PATIENT POPULATION
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL June 21, 2022 NEW PATIENT POPULATION
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX TABLET ORAL Feb. 12, 2023 NEW CHEMICAL ENTITY
75MG;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX TABLET ORAL Feb. 12, 2023 NEW CHEMICAL ENTITY
150MG;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX 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;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX 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;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX 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;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX 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;150MG, 100MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 Feb. 12, 2018 RX 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 ADDITIONAL 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;75MG, 50MG SYMDEKO (COPACKAGED) VERTEX PHARMS INC N210491 June 21, 2019 RX 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 ADDITIONAL 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 EC50 6.57 CHEMBL DRUG LABEL

External reference:

IDSource
8RW88Y506K UNII
4037327 VANDF
C4519194 UMLSCUI
CV6 PDB_CHEM_ID
CHEMBL3544914 ChEMBL_ID
46199646 PUBCHEM_CID
DB11712 DRUGBANK_ID
D11041 KEGG_DRUG
C000625213 MESH_SUPPLEMENTAL_RECORD_UI
10199 IUPHAR_LIGAND_ID
10104 INN_ID
1999382 RXNORM
017465 NDDF
771596001 SNOMEDCT_US
33763 MMSL
d08742 MMSL

Pharmaceutical products:

None