Elexacaftor

Last updated
Elexacaftor
Elexacaftor.svg
Clinical data
Trade names Trikafta and Kaftrio (with ivacaftor and tezacaftor)
Other namesVX-445
AHFS/Drugs.com Monograph
MedlinePlus a619061
License data
Routes of
administration 		By mouth
ATC code
  • None
Legal status
Legal status
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
PubChem CID
DrugBank
ChemSpider
UNII
KEGG
ChEMBL
CompTox Dashboard (EPA)
Chemical and physical data
Formula C26H34F3N7O4S
Molar mass 597.66 g·mol−1
3D model (JSmol)
  • Cc1nn(C)cc1S(=O)(=O)NC(=O)c1ccc(-n2ccc(OCC(C)(C)C(F)(F)F)n2)nc1N1C[C@@H](C)CC1(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

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

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. 1 2 3 "Trikafta (elexacaftor, ivacaftor and tezacaftor) Patient Information". Drugs.com. October 23, 2019. Archived 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. Archived from the original on November 13, 2019. Retrieved November 13, 2019.PD-icon.svg 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. Archived from the original on November 20, 2019. Retrieved November 20, 2019.PD-icon.svg 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.
This page is based on this Wikipedia article
Text is available under the CC BY-SA 4.0 license; additional terms may apply.
Images, videos and audio are available under their respective licenses.