Elexacaftor

Elexacaftor
Clinical data
Trade names	Trikafta and Kaftrio (with ivacaftor and tezacaftor)
Other names	VX-445
AHFS/Drugs.com	Monograph
MedlinePlus	a619061
License data	EU EMA: by INN; US DailyMed: Elexacaftor;
Routes of; administration	By mouth
ATC code	None;
Legal status
Legal status	AU: S4 (Prescription only); US: ℞-only; EU: Rx-only;
Identifiers
	IUPAC 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-carboxamide;
CAS Number	2216712-66-0 ;
PubChem CID	134587348;
DrugBank	DB15444;
ChemSpider	75531299;
UNII	RRN67GMB0V;
KEGG	D11507;
ChEMBL	ChEMBL4298128;
Chemical and physical data
Formula	C26H34F3N7O4S
Molar mass	597.66 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES 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 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.

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. This combination was approved for medical use in the United States in 2019.

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.

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