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Wikipedia

Elexacaftor

Elexacaftor is a medication that acts as cystic fibrosis transmembrane conductance regulator (CFTR) corrector.[1]

Elexacaftor
Clinical data
Trade namesTrikafta and Kaftrio (with ivacaftor and tezacaftor)
Other namesVX-445
AHFS/Drugs.comMonograph
MedlinePlusa619061
License data
Routes of
administration
By mouth
ATC code
  • None
Legal status
Legal status
  • AU: S4 (Prescription only)
  • US: ℞-only
  • EU: Rx-only
Identifiers
  • N-(1,3-dimethylpyrazol-4-yl)sulfonyl-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide
CAS Number
  • 2216712-66-0 Y
PubChem CID
  • 134587348
DrugBank
  • DB15444
ChemSpider
  • 75531299
UNII
  • RRN67GMB0V
KEGG
  • D11507
ChEMBL
  • ChEMBL4298128
Chemical and physical data
FormulaC26H34F3N7O4S
Molar mass597.66 g·mol−1
3D model (JSmol)
  • Interactive image
  • C[C@H]1CC(N(C1)C2=C(C=CC(=N2)N3C=CC(=N3)OCC(C)(C)C(F)(F)F)C(=O)NS(=O)(=O)C4=CN(N=C4C)C)(C)C
  • InChI=1S/C26H34F3N7O4S/c1-16-12-25(5,6)35(13-16)22-18(23(37)33-41(38,39)19-14-34(7)31-17(19)2)8-9-20(30-22)36-11-10-21(32-36)40-15-24(3,4)26(27,28)29/h8-11,14,16H,12-13,15H2,1-7H3,(H,33,37)/t16-/m0/s1
  • Key:MVRHVFSOIWFBTE-INIZCTEOSA-N

It is available in a single pill with ivacaftor and tezacaftor; the fixed-dose combination, elexacaftor/tezacaftor/ivacaftor (brand name Trikafta), is used to treat people with cystic fibrosis who are homozygous for the f508del mutation.[1][2] This combination was approved for medical use in the United States in 2019.[1][3][4]

The fixed-dose combination elexacaftor/tezacaftor/ivacaftor (Kaftrio) was approved for medical use in the European Union in August 2020, for the treatment of cystic fibrosis.[5][6]

References

  1. ^ a b c "Trikafta (elexacaftor, ivacaftor and tezacaftor) Patient Information". Drugs.com. October 23, 2019. from the original on October 30, 2019. Retrieved November 13, 2019.
  2. ^ Ridley K, Condren M (2020). "Elexacaftor-Tezacaftor-Ivacaftor: The First Triple-Combination Cystic Fibrosis Transmembrane Conductance Regulator Modulating Therapy". The Journal of Pediatric Pharmacology and Therapeutics. 25 (3): 192–197. doi:10.5863/1551-6776-25.3.192. PMC 7134581. PMID 32265602.
  3. ^ "FDA approves new breakthrough therapy for cystic fibrosis". U.S. Food and Drug Administration (FDA) (Press release). October 21, 2019. from the original on November 13, 2019. Retrieved November 13, 2019.   This article incorporates text from this source, which is in the public domain.
  4. ^ "Drug Trials Snapshots: Trikafta". U.S. Food and Drug Administration (FDA). October 31, 2019. from the original on November 20, 2019. Retrieved November 20, 2019.   This article incorporates text from this source, which is in the public domain.
  5. ^ "Kaftrio EPAR". European Medicines Agency (EMA). 23 June 2020. Retrieved 24 September 2020.
  6. ^ "New medicine for cystic fibrosis patients". European Medicines Agency (EMA) (Press release). 26 June 2020. Retrieved 26 June 2020.

External links

  • "Elexacaftor". Drug Information Portal. U.S. National Library of Medicine.


elexacaftor, medication, that, acts, cystic, fibrosis, transmembrane, conductance, regulator, cftr, corrector, clinical, datatrade, namestrikafta, kaftrio, with, ivacaftor, tezacaftor, other, namesvx, 445ahfs, drugs, commonographmedlineplusa619061license, data. Elexacaftor is a medication that acts as cystic fibrosis transmembrane conductance regulator CFTR corrector 1 ElexacaftorClinical dataTrade namesTrikafta and Kaftrio with ivacaftor and tezacaftor Other namesVX 445AHFS Drugs comMonographMedlinePlusa619061License dataEU EMA by INN US DailyMed ElexacaftorRoutes ofadministrationBy mouthATC codeNoneLegal statusLegal statusAU S4 Prescription only US only EU Rx onlyIdentifiersIUPAC name N 1 3 dimethylpyrazol 4 yl sulfonyl 6 3 3 3 3 trifluoro 2 2 dimethylpropoxy pyrazol 1 yl 2 4S 2 2 4 trimethylpyrrolidin 1 yl pyridine 3 carboxamideCAS Number2216712 66 0 YPubChem CID134587348DrugBankDB15444ChemSpider75531299UNIIRRN67GMB0VKEGGD11507ChEMBLChEMBL4298128Chemical and physical dataFormulaC 26H 34F 3N 7O 4SMolar mass597 66 g mol 13D model JSmol Interactive imageSMILES C C H 1CC N C1 C2 C C CC N2 N3C CC N3 OCC C C C F F F C O NS O O C4 CN N C4C C C CInChI InChI 1S C26H34F3N7O4S c1 16 12 25 5 6 35 13 16 22 18 23 37 33 41 38 39 19 14 34 7 31 17 19 2 8 9 20 30 22 36 11 10 21 32 36 40 15 24 3 4 26 27 28 29 h8 11 14 16H 12 13 15H2 1 7H3 H 33 37 t16 m0 s1Key MVRHVFSOIWFBTE INIZCTEOSA NIt is available in a single pill with ivacaftor and tezacaftor the fixed dose combination elexacaftor tezacaftor ivacaftor brand name Trikafta is used to treat people with cystic fibrosis who are homozygous for the f508del mutation 1 2 This combination was approved for medical use in the United States in 2019 1 3 4 The fixed dose combination elexacaftor tezacaftor ivacaftor Kaftrio was approved for medical use in the European Union in August 2020 for the treatment of cystic fibrosis 5 6 References Edit a b c Trikafta elexacaftor ivacaftor and tezacaftor Patient Information Drugs com October 23 2019 Archived from the original on October 30 2019 Retrieved November 13 2019 Ridley K Condren M 2020 Elexacaftor Tezacaftor Ivacaftor The First Triple Combination Cystic Fibrosis Transmembrane Conductance Regulator Modulating Therapy The Journal of Pediatric Pharmacology and Therapeutics 25 3 192 197 doi 10 5863 1551 6776 25 3 192 PMC 7134581 PMID 32265602 FDA approves new breakthrough therapy for cystic fibrosis U S Food and Drug Administration FDA Press release October 21 2019 Archived from the original on November 13 2019 Retrieved November 13 2019 This article incorporates text from this source which is in the public domain Drug Trials Snapshots Trikafta U S Food and Drug Administration FDA October 31 2019 Archived from the original on November 20 2019 Retrieved November 20 2019 This article incorporates text from this source which is in the public domain Kaftrio EPAR European Medicines Agency EMA 23 June 2020 Retrieved 24 September 2020 New medicine for cystic fibrosis patients European Medicines Agency EMA Press release 26 June 2020 Retrieved 26 June 2020 External links Edit Elexacaftor Drug Information Portal U S National Library of Medicine Portal Medicine This drug article relating to the respiratory system is a stub You can help Wikipedia by expanding it vte Retrieved from https en wikipedia org w index php title Elexacaftor amp oldid 1136348330, wikipedia, wiki, book, books, library,

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