Tezacaftor

Tezacaftor
Clinical data
Trade names	Symdeko (with ivacaftor)
Identifiers
	IUPAC name 1-(2,2-Difluoro-1,3-benzodioxol-5-yl)-N-[1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(2-hydroxy-1,1-dimethylethyl)-1H-indol-5-yl]-cyclopropanecarboxamide;
CAS Number	1152311-62-0;
PubChem CID	46199646;
DrugBank	DB11712;
UNII	8RW88Y506K;
KEGG	D11041;
Chemical and physical data
Formula	C26H27F3N2O6
Molar mass	520.51 g·mol−1

Tezacaftor, also known as VX-661, is a drug approved by the FDA to treat some cases of cystic fibrosis.

In 2018, the FDA approved a combination of ivacaftor and tezacaftor; the manufacturer announced a list price of $292,000 per year.^[1] The combination of ivacaftor and tezacaftor is being sold as Symdeko.^[2]

Mechanism of action

Tezacaftor helps move the CFTR protein to the correct position on the cell surface, and is designed to treat people with the F508del mutation.^[3]

Properties

Its chemical formula is C₂₆H₂₇F₃N₂O₆, with a molecular weight of 520.505 g/mol.^[4]

Clinical trials

The EVOLVE and EXPAND study findings were published in 2017.^[2]

References

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[1] FDA approves another Vertex drug for treatment of cystic fibrosis

[mp-fda-2] 1 2 FDA Approves New Cystic Fibrosis Drug Combo - Symdeko approved for patients with specific CFTR mutations Feb 2018

[3] Tezacaftor (VX-661) for Cystic Fibrosis

[pubchem-4] Tezacaftor includes structure