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

Stem definitionDrug idCAS RN
cystic fibrosis transmembrane regulator (CFTR) protein modulators 4228 873054-44-5

Description:

MoleculeDescription

Molfile Inchi Smiles

Synonyms:

  • kalydeco
  • VX 770
  • ivacaftor
Ivacaftor is a potentiator of the CFTR protein. The CFTR protein is a chloride channel present at the surface of epithelial cells in multiple organs. Ivacaftor facilitates increased chloride transport by potentiating the channel-open probability (or gating) of the CFTR protein.
  • Molecular weight: 392.50
  • Formula: C24H28N2O3
  • CLOGP: 3.82
  • LIPINSKI: 0
  • HAC: 5
  • HDO: 3
  • TPSA: 78.43
  • ALOGS: -5.29
  • ROTB: 4

  • Status: ONP

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

Drug dosage:

DoseUnitRoute
0.30 g O

ADMET properties:

PropertyValueReference
BDDCS (Biopharmaceutical Drug Disposition Classification System) 2 Hosey CM, Chan R, Benet LZ

Approvals:

DateAgencyCompanyOrphan
Aug. 21, 2020 EMA Vertex Pharmaceuticals (Ireland) Limited
Jan. 31, 2012 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 6354.94 33.92 1225 10287 9582 46664968
Hospitalisation 1099.59 33.92 454 11058 62931 46611619
Cystic fibrosis 924.75 33.92 208 11304 3480 46671070
Pulmonary function test decreased 527.07 33.92 133 11379 3687 46670863
Cystic fibrosis respiratory infection suppression 463.58 33.92 83 11429 384 46674166
Infection 378.83 33.92 291 11221 133301 46541249
Haemoptysis 304.12 33.92 142 11370 26047 46648503
Cough 224.14 33.92 279 11233 229970 46444580
Forced expiratory volume decreased 184.95 33.92 63 11449 5041 46669509
Productive cough 178.14 33.92 126 11386 50589 46623961
Influenza 152.92 33.92 146 11366 89124 46585426
Sputum increased 141.54 33.92 40 11472 1716 46672834
Respiration abnormal 137.59 33.92 49 11463 4472 46670078
Chest discomfort 125.75 33.92 132 11380 90137 46584413
Distal intestinal obstruction syndrome 124.86 33.92 27 11485 370 46674180
Pneumonia 118.01 33.92 275 11237 376045 46298505
Abdominal pain upper 112.93 33.92 158 11354 145147 46529403
Pseudomonas infection 110.32 33.92 52 11460 9706 46664844
Headache 97.57 33.92 298 11214 478054 46196496
Drug ineffective 70.65 33.92 40 11472 677798 45996752
Cystic fibrosis lung 70.62 33.92 14 11498 121 46674429
Treatment noncompliance 69.41 33.92 58 11454 29673 46644877
Pneumonia pseudomonal 64.73 33.92 23 11489 2083 46672467
Emergency care 62.55 33.92 18 11494 824 46673726
Lung transplant 61.13 33.92 24 11488 2868 46671682
Sinusitis 53.20 33.92 106 11406 129662 46544888
Sinus operation 52.85 33.92 18 11494 1437 46673113
Anaemia 51.09 33.92 3 11509 255776 46418774
Viral infection 50.79 33.92 49 11463 30186 46644364
Nasopharyngitis 50.66 33.92 115 11397 153883 46520667
Constipation 48.63 33.92 122 11390 173975 46500575
Respiratory tract congestion 47.73 33.92 33 11479 12731 46661819
Rhinovirus infection 46.14 33.92 21 11491 3615 46670935
Cystic fibrosis related diabetes 43.98 33.92 8 11504 41 46674509
Paranasal sinus hypersecretion 42.11 33.92 19 11493 3205 46671345
Insurance issue 42.00 33.92 16 11496 1762 46672788
Sinus disorder 37.99 33.92 32 11480 16531 46658019
Hypotension 37.92 33.92 6 11506 232583 46441967
Pneumothorax 37.19 33.92 30 11482 14599 46659951
Acute kidney injury 36.45 33.92 7 11505 235848 46438702
Pain 35.83 33.92 39 11473 476909 46197641
Pharyngitis streptococcal 34.29 33.92 23 11489 8445 46666105

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 4428.03 37.46 897 7855 7675 29936051
Hospitalisation 1032.19 37.46 411 8341 43908 29899818
Cystic fibrosis 659.43 37.46 149 8603 2125 29941601
Cystic fibrosis respiratory infection suppression 431.50 37.46 78 8674 313 29943413
Pulmonary function test decreased 370.59 37.46 107 8645 4188 29939538
Infection 222.33 37.46 183 8569 77891 29865835
Cough 208.95 37.46 218 8534 125424 29818302
Productive cough 208.93 37.46 122 8630 29831 29913895
Influenza 193.10 37.46 133 8619 43265 29900461
Forced expiratory volume decreased 173.44 37.46 56 8696 3194 29940532
Abdominal pain upper 150.00 37.46 133 8619 62418 29881308
Lung transplant 131.41 37.46 40 8712 1876 29941850
Nasopharyngitis 130.94 37.46 120 8632 58729 29884997
Respiration abnormal 126.72 37.46 42 8710 2600 29941126
Sputum increased 119.84 37.46 37 8715 1820 29941906
Haemoptysis 114.51 37.46 85 8667 30993 29912733
Headache 109.85 37.46 193 8559 182113 29761613
Chest discomfort 101.60 37.46 93 8659 45387 29898339
Liver function test increased 84.23 37.46 48 8704 11168 29932558
Product dose omission issue 76.92 37.46 113 8639 91518 29852208
Distal intestinal obstruction syndrome 74.44 37.46 18 8734 347 29943379
Pneumonia 71.87 37.46 237 8515 334069 29609657
Sinusitis 68.28 37.46 65 8687 33280 29910446
Insurance issue 63.94 37.46 19 8733 819 29942907
Sinus operation 63.70 37.46 18 8734 648 29943078
Pseudomonas infection 63.36 37.46 39 8713 10443 29933283
Cystic fibrosis lung 61.38 37.46 12 8740 79 29943647
Hepatic enzyme increased 55.13 37.46 58 8694 33436 29910290
Treatment noncompliance 51.25 37.46 49 8703 25204 29918522
Testicular pain 44.96 37.46 20 8732 2752 29940974
Increased bronchial secretion 43.55 37.46 16 8736 1343 29942383
Anaemia 43.46 37.46 5 8747 207987 29735739
Respiratory tract congestion 42.50 37.46 24 8728 5482 29938244
Hypotension 41.46 37.46 5 8747 200560 29743166
Sinus disorder 39.76 37.46 24 8728 6201 29937525
Paranasal sinus hypersecretion 39.61 37.46 13 8739 779 29942947
Acute kidney injury 38.50 37.46 16 8736 273826 29669900

Pharmacologic Action:

SourceCodeDescription
ATC R07AX02 RESPIRATORY SYSTEM
OTHER RESPIRATORY SYSTEM PRODUCTS
OTHER RESPIRATORY SYSTEM PRODUCTS
Other respiratory system products
ATC R07AX30 RESPIRATORY SYSTEM
OTHER RESPIRATORY SYSTEM PRODUCTS
OTHER RESPIRATORY SYSTEM PRODUCTS
Other respiratory system products
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
MeSH PA D065101 Chloride Channel Agonists
MeSH PA D049990 Membrane Transport Modulators
FDA MoA N0000182141 Cytochrome P450 3A4 Inhibitors
FDA MoA N0000184145 Chloride Channel Activation Potentiators
FDA EPC N0000184146 Cystic Fibrosis Transmembrane Conductance Regulator Potentiator
FDA MoA N0000185503 P-Glycoprotein Inhibitors
FDA MoA N0000185504 Cytochrome P450 2C9 Inhibitors
FDA MoA N0000190114 Cytochrome P450 3A Inhibitors
CHEBI has role CHEBI:66902 cftr potentiators
CHEBI has role CHEBI:71031 orphan drugs

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 8.08 acidic
pKa2 9.12 acidic
pKa3 11.19 acidic

Orange Book patent data (new drug applications)

None

Orange Book exclusivity data (new drug applications)

Formulation strengthTrade nameApplicantApplication numberApproval dateTypeDose formRouteExclusivity dateDescription
125MG;100MG ORKAMBI VERTEX PHARMS INC N206038 Sept. 28, 2016 RX TABLET ORAL Jan. 25, 2021 ADDITIONULLL INFORMATION ADDED TO THE PEDIATRIC USE SECTION OF THE LABELING REGARDING A NEW CLINICAL TRIAL IN PATIENTS AGED 6 THROUGH 11 YEARS (TRIAL 4)
125MG;200MG ORKAMBI VERTEX PHARMS INC N206038 July 2, 2015 RX TABLET ORAL Jan. 25, 2021 ADDITIONULLL INFORMATION ADDED TO THE PEDIATRIC USE SECTION OF THE LABELING REGARDING A NEW CLINICAL TRIAL IN PATIENTS AGED 6 THROUGH 11 YEARS (TRIAL 4)
150MG KALYDECO VERTEX PHARMS N203188 Jan. 31, 2012 RX TABLET ORAL Feb. 21, 2021 TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 6 YEARS AND OLDER WHO HAVE ONE OF THE FOLLOWING MUTATIONS IN THE CFTR GENE: G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, OR S549R
125MG/PACKET;100MG/PACKET ORKAMBI VERTEX PHARMS INC N211358 Aug. 7, 2018 RX GRANULE ORAL Aug. 7, 2021 NEW PRODUCT
188MG/PACKET;150MG/PACKET ORKAMBI VERTEX PHARMS INC N211358 Aug. 7, 2018 RX GRANULE ORAL Aug. 7, 2021 NEW PRODUCT
150MG KALYDECO VERTEX PHARMS N203188 Jan. 31, 2012 RX TABLET ORAL Dec. 29, 2021 TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 6 YEARS AND OLDER WHO HAVE AN R117H MUTATION IN THE CFTR GENE
25MG/PACKET KALYDECO VERTEX PHARMS INC N207925 April 29, 2019 RX GRANULE ORAL March 17, 2022 TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGES 2 TO LESS THAN 6 YEARS WHO HAVE ONE OF THE FOLLOWING MUTATIONS IN THE CFTR GENE: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, AND R117H
50MG/PACKET KALYDECO VERTEX PHARMS INC N207925 March 17, 2015 RX GRANULE ORAL March 17, 2022 TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGES 2 TO LESS THAN 6 YEARS WHO HAVE ONE OF THE FOLLOWING MUTATIONS IN THE CFTR GENE: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, AND R117H
75MG/PACKET KALYDECO VERTEX PHARMS INC N207925 March 17, 2015 RX GRANULE ORAL March 17, 2022 TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGES 2 TO LESS THAN 6 YEARS WHO HAVE ONE OF THE FOLLOWING MUTATIONS IN THE CFTR GENE: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, AND R117H
25MG/PACKET KALYDECO VERTEX PHARMS INC N207925 April 29, 2019 RX GRANULE ORAL April 29, 2022 NEW PATIENT POPULATION
50MG/PACKET KALYDECO VERTEX PHARMS INC N207925 March 17, 2015 RX GRANULE ORAL April 29, 2022 NEW PATIENT POPULATION
75MG/PACKET KALYDECO VERTEX PHARMS INC N207925 March 17, 2015 RX GRANULE ORAL April 29, 2022 NEW PATIENT POPULATION
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
125MG;100MG ORKAMBI VERTEX PHARMS INC N206038 Sept. 28, 2016 RX TABLET ORAL July 2, 2022 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 12 YEARS AND OLDER WHO ARE HOMOZYGOUS FOR F508DEL MUTATION IN THE CFTR GENE
125MG;200MG ORKAMBI VERTEX PHARMS INC N206038 July 2, 2015 RX TABLET ORAL July 2, 2022 TREATMENT OF CYSTIC FIBROSIS IN PATIENTS AGE 12 YEARS AND OLDER WHO ARE HOMOZYGOUS FOR F508DEL MUTATION IN THE CFTR GENE
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
100MG,75MG,50MG;N/A,150MG,N/A TRIKAFTA (COPACKAGED) VERTEX PHARMS INC N212273 Oct. 21, 2019 RX TABLET, TABLET ORAL Feb. 12, 2023 NEW CHEMICAL ENTITY
125MG;100MG ORKAMBI VERTEX PHARMS INC N206038 Sept. 28, 2016 RX TABLET ORAL Sept. 28, 2023 TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 6-11 YEAR OLD WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE
125MG;200MG ORKAMBI VERTEX PHARMS INC N206038 July 2, 2015 RX TABLET ORAL Sept. 28, 2023 TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 6-11 YEAR OLD WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE
150MG KALYDECO VERTEX PHARMS N203188 Jan. 31, 2012 RX TABLET ORAL May 17, 2024 TX OF CF IN PTS 2 YRS AND OLDER WHO HAVE ONE OF THE FOLLOWING MUTATIONS IN THE CFTR GENE: E56K, P67L, R74W, D110E, D110H, R117C, E193K, L206W, R347H, R352Q, A455E, D579G, S945L, S977F, F1052V, K1060T, A1067T, G1069R, R1070Q, R1070W, F1074L, D1152H, D1270N
25MG/PACKET KALYDECO VERTEX PHARMS INC N207925 April 29, 2019 RX GRANULE ORAL May 17, 2024 TX OF CF IN PTS 2 YRS AND OLDER WHO HAVE ONE OF THE FOLLOWING MUTATIONS IN THE CFTR GENE: E56K, P67L, R74W, D110E, D110H, R117C, E193K, L206W, R347H, R352Q, A455E, D579G, S945L, S977F, F1052V, K1060T, A1067T, G1069R, R1070Q, R1070W, F1074L, D1152H, D1270N
50MG/PACKET KALYDECO VERTEX PHARMS INC N207925 March 17, 2015 RX GRANULE ORAL May 17, 2024 TX OF CF IN PTS 2 YRS AND OLDER WHO HAVE ONE OF THE FOLLOWING MUTATIONS IN THE CFTR GENE: E56K, P67L, R74W, D110E, D110H, R117C, E193K, L206W, R347H, R352Q, A455E, D579G, S945L, S977F, F1052V, K1060T, A1067T, G1069R, R1070Q, R1070W, F1074L, D1152H, D1270N
75MG/PACKET KALYDECO VERTEX PHARMS INC N207925 March 17, 2015 RX GRANULE ORAL May 17, 2024 TX OF CF IN PTS 2 YRS AND OLDER WHO HAVE ONE OF THE FOLLOWING MUTATIONS IN THE CFTR GENE: E56K, P67L, R74W, D110E, D110H, R117C, E193K, L206W, R347H, R352Q, A455E, D579G, S945L, S977F, F1052V, K1060T, A1067T, G1069R, R1070Q, R1070W, F1074L, D1152H, D1270N
150MG KALYDECO VERTEX PHARMS N203188 Jan. 31, 2012 RX TABLET ORAL July 31, 2024 TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 2 YEARS AND OLDER WHO HAVE ONE OF THE FOLLOWING MUTATIONS IN THE CFTR GENE: 711+3A-G, E831X, 2789+5G-A, 3272-26A-G, AND 3849+10KBC-T
25MG/PACKET KALYDECO VERTEX PHARMS INC N207925 April 29, 2019 RX GRANULE ORAL July 31, 2024 TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 2 YEARS AND OLDER WHO HAVE ONE OF THE FOLLOWING MUTATIONS IN THE CFTR GENE: 711+3A-G, E831X, 2789+5G-A, 3272-26A-G, AND 3849+10KBC-T
50MG/PACKET KALYDECO VERTEX PHARMS INC N207925 March 17, 2015 RX GRANULE ORAL July 31, 2024 TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 2 YEARS AND OLDER WHO HAVE ONE OF THE FOLLOWING MUTATIONS IN THE CFTR GENE: 711+3A-G, E831X, 2789+5G-A, 3272-26A-G, AND 3849+10KBC-T
75MG/PACKET KALYDECO VERTEX PHARMS INC N207925 March 17, 2015 RX GRANULE ORAL July 31, 2024 TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 2 YEARS AND OLDER WHO HAVE ONE OF THE FOLLOWING MUTATIONS IN THE CFTR GENE: 711+3A-G, E831X, 2789+5G-A, 3272-26A-G, AND 3849+10KBC-T
100MG,75MG,50MG;N/A,150MG,N/A TRIKAFTA (COPACKAGED) VERTEX PHARMS INC N212273 Oct. 21, 2019 RX TABLET, TABLET ORAL Oct. 21, 2024 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
125MG/PACKET;100MG/PACKET ORKAMBI VERTEX PHARMS INC N211358 Aug. 7, 2018 RX GRANULE ORAL Aug. 7, 2025 FOR THE TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 2 THROUGH 5 YEARS OLD WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE
188MG/PACKET;150MG/PACKET ORKAMBI VERTEX PHARMS INC N211358 Aug. 7, 2018 RX GRANULE ORAL Aug. 7, 2025 FOR THE TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 2 THROUGH 5 YEARS OLD WHO ARE HOMOZYGOUS FOR THE F508DEL MUTATION IN THE CFTR GENE
150MG KALYDECO VERTEX PHARMS N203188 Jan. 31, 2012 RX TABLET ORAL Aug. 15, 2025 THE TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 12 MONTHS AND OLDER WHO HAVE ONE MUTATION IN THE CFTR GENE THAT IS RESPONSIVE TO IVACAFTOR POTENTIATION BASED ON CLINICAL AND/OR IN VITRO ASSAY DATA
25MG/PACKET KALYDECO VERTEX PHARMS INC N207925 April 29, 2019 RX GRANULE ORAL Aug. 15, 2025 THE TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 12 MONTHS AND OLDER WHO HAVE ONE MUTATION IN THE CFTR GENE THAT IS RESPONSIVE TO IVACAFTOR POTENTIATION BASED ON CLINICAL AND/OR IN VITRO ASSAY DATA
50MG/PACKET KALYDECO VERTEX PHARMS INC N207925 March 17, 2015 RX GRANULE ORAL Aug. 15, 2025 THE TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 12 MONTHS AND OLDER WHO HAVE ONE MUTATION IN THE CFTR GENE THAT IS RESPONSIVE TO IVACAFTOR POTENTIATION BASED ON CLINICAL AND/OR IN VITRO ASSAY DATA
75MG/PACKET KALYDECO VERTEX PHARMS INC N207925 March 17, 2015 RX GRANULE ORAL Aug. 15, 2025 THE TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 12 MONTHS AND OLDER WHO HAVE ONE MUTATION IN THE CFTR GENE THAT IS RESPONSIVE TO IVACAFTOR POTENTIATION BASED ON CLINICAL AND/OR IN VITRO ASSAY DATA
25MG/PACKET KALYDECO VERTEX PHARMS INC N207925 April 29, 2019 RX GRANULE ORAL April 29, 2026 TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 6 MONTHS TO LESS THAN 12 MONTHS WHO HAVE ONE MUTATION IN THE CFTR GENE THAT IS RESPONSIVE TO IVACAFTOR POTENTIATION BASED ON CLINICAL AND/OR IN VITRO ASSAY DATA
50MG/PACKET KALYDECO VERTEX PHARMS INC N207925 March 17, 2015 RX GRANULE ORAL April 29, 2026 TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 6 MONTHS TO LESS THAN 12 MONTHS WHO HAVE ONE MUTATION IN THE CFTR GENE THAT IS RESPONSIVE TO IVACAFTOR POTENTIATION BASED ON CLINICAL AND/OR IN VITRO ASSAY DATA
75MG/PACKET KALYDECO VERTEX PHARMS INC N207925 March 17, 2015 RX GRANULE ORAL April 29, 2026 TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 6 MONTHS TO LESS THAN 12 MONTHS WHO HAVE ONE MUTATION IN THE CFTR GENE THAT IS RESPONSIVE TO IVACAFTOR POTENTIATION BASED ON CLINICAL AND/OR IN VITRO ASSAY DATA
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
100MG,75MG,50MG;N/A,150MG,N/A TRIKAFTA (COPACKAGED) VERTEX PHARMS INC N212273 Oct. 21, 2019 RX TABLET, TABLET ORAL Oct. 21, 2026 INDICATED FOR THE TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGED 12 YEARS AND OLDER WHO HAVE AT LEAST ONE F508DEL MUTATION IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE
150MG KALYDECO VERTEX PHARMS N203188 Jan. 31, 2012 RX TABLET ORAL Dec. 21, 2027 FOR THE TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 4 MONTHS AND OLDER WHO HAVE ONE OF THE ADDITIONULLL MUTATIONS IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE THAT HAVE BEEN IDENTIFIED AS RESPONSIVE TO IVACAFTOR POTENTIATION BASED ON IN VITRO DATA AND IDENTIFIED IN THE APPROVAL ON DECEMBER 21, 2020
25MG/PACKET KALYDECO VERTEX PHARMS INC N207925 April 29, 2019 RX GRANULE ORAL Dec. 21, 2027 FOR THE TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 4 MONTHS AND OLDER WHO HAVE ONE OF THE ADDITIONULLL MUTATIONS IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE THAT HAVE BEEN IDENTIFIED AS RESPONSIVE TO IVACAFTOR POTENTIATION BASED ON IN VITRO DATA AND IDENTIFIED IN THE APPROVAL ON DECEMBER 21, 2020
50MG/PACKET KALYDECO VERTEX PHARMS INC N207925 March 17, 2015 RX GRANULE ORAL Dec. 21, 2027 FOR THE TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 4 MONTHS AND OLDER WHO HAVE ONE OF THE ADDITIONULLL MUTATIONS IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE THAT HAVE BEEN IDENTIFIED AS RESPONSIVE TO IVACAFTOR POTENTIATION BASED ON IN VITRO DATA AND IDENTIFIED IN THE APPROVAL ON DECEMBER 21, 2020
75MG/PACKET KALYDECO VERTEX PHARMS INC N207925 March 17, 2015 RX GRANULE ORAL Dec. 21, 2027 FOR THE TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGE 4 MONTHS AND OLDER WHO HAVE ONE OF THE ADDITIONULLL MUTATIONS IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE THAT HAVE BEEN IDENTIFIED AS RESPONSIVE TO IVACAFTOR POTENTIATION BASED ON IN VITRO DATA AND IDENTIFIED IN THE APPROVAL ON DECEMBER 21, 2020
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
100MG,75MG,50MG;N/A,150MG,N/A TRIKAFTA (COPACKAGED) VERTEX PHARMS INC N212273 Oct. 21, 2019 RX TABLET, TABLET ORAL Dec. 21, 2027 TREATMENT OF CYSTIC FIBROSIS (CF) IN PATIENTS AGED 12 YEARS AND OLDER WHO HAVE A MUTATION IN THE CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR (CFTR) GENE THAT IS RESPONSIVE BASED ON IN VITRO DATA

Bioactivity Summary:

TargetClassPharosUniProtActionTypeActivity value
(-log[M])
Mechanism
action
Bioact sourceMoA source
Cystic fibrosis transmembrane conductance regulator Ion channel POSITIVE MODULATOR EC50 8.52 CHEMBL CHEMBL

External reference:

IDSource
D09916 KEGG_DRUG
4031568 VANDF
C3264621 UMLSCUI
CHEBI:66901 CHEBI
VX7 PDB_CHEM_ID
CHEMBL2010601 ChEMBL_ID
DB08820 DRUGBANK_ID
C545203 MESH_SUPPLEMENTAL_RECORD_UI
4342 IUPHAR_LIGAND_ID
9263 INN_ID
1Y740ILL1Z UNII
16220172 PUBCHEM_CID
1243041 RXNORM
187124 MMSL
28301 MMSL
d07826 MMSL
014156 NDDF
703823007 SNOMEDCT_US
703824001 SNOMEDCT_US

Pharmaceutical products:

ProductCategoryIngredientsNDCFormQuantityRouteMarketingLabel
Kalydeco HUMAN PRESCRIPTION DRUG LABEL 1 51167-200 TABLET, FILM COATED 150 mg ORAL NDA 28 sections
Kalydeco HUMAN PRESCRIPTION DRUG LABEL 1 51167-300 GRANULE 50 mg ORAL NDA 28 sections
Kalydeco HUMAN PRESCRIPTION DRUG LABEL 1 51167-400 GRANULE 75 mg ORAL NDA 28 sections
ORKAMBI HUMAN PRESCRIPTION DRUG LABEL 2 51167-500 GRANULE 188 mg ORAL NDA 30 sections
Kalydeco HUMAN PRESCRIPTION DRUG LABEL 1 51167-600 GRANULE 25 mg ORAL NDA 28 sections
ORKAMBI HUMAN PRESCRIPTION DRUG LABEL 2 51167-700 TABLET, FILM COATED 125 mg ORAL NDA 30 sections
ORKAMBI HUMAN PRESCRIPTION DRUG LABEL 2 51167-809 TABLET, FILM COATED 125 mg ORAL NDA 30 sections
ORKAMBI HUMAN PRESCRIPTION DRUG LABEL 2 51167-900 GRANULE 125 mg ORAL NDA 30 sections