Elexacaftor

Elexacaftor is a pharmaceutical drug 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
License data	US DailyMed: Elexacaftor
Routes of administration	By mouth
ATC code	(combination with ivacaftor and tezacaftor)
Legal status
Legal status	US: ℞-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
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

It is available in a single pill with ivacaftor and tezacaftor; the combination, Elexacaftor/tezacaftor/ivacaftor (brand name Trikafta), is used to treat people with cystic fibrosis who are homozygous for the f508del mutation.[1] This combination was approved for medical use in the United States in 2019.[1][2][3] The list price in the US is expected to be $311,000 per year.[4]

In June 2020, the Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA) recommended the approval of elexacaftor/tezacaftor/ivacaftor (Kaftrio) for the treatment of cystic fibrosis.[5]

References

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.
2. "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.
3. "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.
4. Maddipatla, Manojna; O'Donnell, Carl (October 21, 2019). "Vertex prices cystic fibrosis combo treatment at $311,000-per-year". Reuters. Retrieved October 23, 2019.
5. "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.