Elexacaftor

Cystic fibrosis medication
  • EU EMA: by INN
  • US DailyMed: Elexacaftor
Routes of
administrationBy mouthATC code
  • None
Legal statusLegal 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 checkY
PubChem CID
  • 134587348
DrugBank
  • DB15444
ChemSpider
  • 75531299
UNII
  • RRN67GMB0V
KEGG
  • D11507
ChEMBL
  • ChEMBL4298128
CompTox Dashboard (EPA)
  • DTXSID901027907 Edit this at Wikidata
Chemical and physical dataFormulaC26H34F3N7O4SMolar mass597.66 g·mol−13D 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

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. ^ 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.
  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. Public Domain 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. Public Domain 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.
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