Elexacaftor information

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;
CompTox Dashboard (EPA)	DTXSID901027907 ;
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.^[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]

^ ^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.

[Drugs2019-1] "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_2020-2] 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.

[FDA_PR-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. This article incorporates text from this source, which is in the public domain.

[FDA_Snapshot-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. This article incorporates text from this source, which is in the public domain.

[Kaftrio_EPAR-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.

Elexacaftor information

and 10 Related for: Elexacaftor information

Elexacaftor

Tezacaftor

Ivacaftor

Cystic fibrosis

ATC code R07

Vertex Pharmaceuticals

List of drugs granted breakthrough therapy designation

Cystic fibrosis and race

Peter Grootenhuis

Paul Negulescu