Tezacaftor

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

Tezacaftor
Clinical data
Trade namesSymdeko (with ivacaftor)
Other namesVX-661
License data
Routes of
administration		By mouth
ATC code
Legal status
Legal status
Identifiers
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
KEGG
CompTox Dashboard (EPA)
Chemical and physical data
FormulaC26H27F3N2O6
Molar mass520.505 g·mol−1

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 for tezacaftor and ivacaftor combination therapy orphan drug designation and priority review; and granted the approval of Symdeko to Vertex Pharmaceuticals Incorporated.[2][6]

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
  6. "Tezacaftor and Ivacaftor Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). 15 June 2017. Retrieved 25 October 2020.


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