Elexacaftor

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

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 Y
PubChem CID	134587348
DrugBank	DB15444
ChemSpider	75531299
UNII	RRN67GMB0V
KEGG	D11507
ChEMBL	ChEMBL4298128
CompTox Dashboard (EPA)	DTXSID901027907
Chemical and physical data
Formula	C26H34F3N7O4S
Molar mass	597.66 g·mol−1
3D model (JSmol)	Interactive image
SMILES 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 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]