Tezacaftor

Tezacaftor, development code VX-661, is a drug that is sold as a fixed-dose combination with ivacaftor as Symdeko. It is used for the treatment of cystic fibrosis (CF) in people six years and older, who have specific gene mutations.[1][2]

Tezacaftor

Clinical data
Trade names	Symdeko (with ivacaftor)
Other names	VX-661
License data	US DailyMed: Tezacaftor
Routes of administration	By mouth
ATC code	R07AX31 (WHO) (combination with ivacaftor)
Legal status
Legal status	US: ℞-only
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
ChemSpider	28637762
UNII	8RW88Y506K
KEGG	D11041
CompTox Dashboard (EPA)	DTXSID40673070
Chemical and physical data
Formula	C26H27F3N2O6
Molar mass	520.505 g·mol^−1
InChI InChI=1S/C26H27F3N2O6/c1-24(2,13-33)22-8-14-7-18(17(27)10-19(14)31(22)11-16(34)12-32)30-23(35)25(5-6-25)15-3-4-20-21(9-15)37-26(28,29)36-20/h3-4,7-10,16,32-34H,5-6,11-13H2,1-2H3,(H,30,35)/t16-/m1/s1 Key:MJUVRTYWUMPBTR-MRXNPFEDSA-N

In 2018, the U.S. Food and Drug Administration (FDA) approved a combination of ivacaftor and tezacaftor;[1] the manufacturer announced a list price of $292,000 per year.[3] The combination of ivacaftor and tezacaftor is being sold as Symdeko.[1][4]

Mechanism of action

Tezacaftor helps move the cystic fibrosis transmembrane conductance regulator (CFTR) protein to the correct position on the cell surface, and is designed to treat people with the F508del mutation.[1][5]

Clinical trials

The EVOLVE and EXPAND study findings were published in 2017.[4]

History

The U.S. Food and Drug Administration (FDA) granted the application priority review and granted the approval of Symdeko to Vertex Pharmaceuticals Incorporated.[2]

References

1. "Drug Trials Snapshots: Symdeko". U.S. Food and Drug Administration. 7 March 2018. Archived from the original on 21 November 2019. Retrieved 20 November 2019. This article incorporates text from this source, which is in the public domain.
2. "FDA expands approval of treatment for cystic fibrosis to include patients ages 6 and older". U.S. Food and Drug Administration (FDA) (Press release). 21 June 2019. Archived from the original on 21 November 2019. Retrieved 20 November 2019. This article incorporates text from this source, which is in the public domain.
3. "FDA approves another Vertex drug for treatment of cystic fibrosis". The Boston Globe. 17 July 2018. Retrieved 20 November 2019.
4. FDA Approves New Cystic Fibrosis Drug Combo - Symdeko approved for patients with specific CFTR mutations Feb 2018
5. Tezacaftor (VX-661) for Cystic Fibrosis

External links

• "Tezacaftor". Drug Information Portal. U.S. National Library of Medicine.
• "Ivacaftor regimen with Tezacaftor". Drug Information Portal. U.S. National Library of Medicine.