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.[3][4][5]

Tezacaftor
Clinical data
Trade namesSymdeko (with ivacaftor)
Other namesVX-661
License data
Routes of
administration		By mouth
ATC code
Legal status
Legal status
Identifiers
  • 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
PubChem CID
DrugBank
ChemSpider
UNII
KEGG
CompTox Dashboard (EPA)
Chemical and physical data
FormulaC26H27F3N2O6
Molar mass520.505 g·mol−1
  • 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.[6] The combination of ivacaftor and tezacaftor is being sold as Symdeko.[1][7]

In 2019, the U.S. Food and Drug Aministration (FDA) approved a combination of elexacaftor, tezacaftor, and ivacaftor;[8] the manufacturer announced a list price of $311,000 per year.[9] The combination of elexacaftor, tezacaftor, and ivacaftor is being sold as Trikafta.[10]

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][11]

Clinical trials

The EVOLVE and EXPAND study findings were published in 2017.[7]

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][12]

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. Lommatzsch ST, Taylor-Cousar JL (2019). "The combination of tezacaftor and ivacaftor in the treatment of patients with cystic fibrosis: clinical evidence and future prospects in cystic fibrosis therapy". Therapeutic Advances in Respiratory Disease. 13: 1753466619844424. doi:10.1177/1753466619844424. PMC 6487765. PMID 31027466.
  4. Paterson SL, Barry PJ, Horsley AR (January 2020). "Tezacaftor and ivacaftor for the treatment of cystic fibrosis". Expert Review of Respiratory Medicine. 14 (1): 15–30. doi:10.1080/17476348.2020.1682998. PMID 31626570.
  5. Guerra L, Favia M, Di Gioia S, Laselva O, Bisogno A, Casavola V, Colombo C, Conese M (August 2020). "The preclinical discovery and development of the combination of ivacaftor + tezacaftor used to treat cystic fibrosis". Expert Opinion on Drug Discovery. 15 (8): 873–891. doi:10.1080/17460441.2020.1750592. PMID 32290721.
  6. "FDA approves another Vertex drug for treatment of cystic fibrosis". The Boston Globe. 17 July 2018. Retrieved 20 November 2019.
  7. Boyles S (13 February 2018). "FDA Approves New Cystic Fibrosis Drug Combo - Symdeko approved for patients with specific CFTR mutations". MedPage Today, LLC.
  8. "FDA approves new breakthrough therapy for cystic fibrosis". U.S. Food and Drug Administration (FDA) (Press release). 21 October 2019. Archived from the original on 13 November 2019. Retrieved 13 November 2019. This article incorporates text from this source, which is in the public domain.
  9. Maddipatla, Manojna; O'Donnell, Carl (21 October 2019). "Vertex prices cystic fibrosis combo treatment at $311,000-per-year". Reuters. Retrieved 23 October 2019.
  10. "Trikafta- elexacaftor, tezacaftor, and ivacaftor kit". DailyMed. 29 January 2020. Retrieved 22 August 2020.
  11. "Tezacaftor (VX-661) for Cystic Fibrosis". Cystic Fibrosis News Today. Pensacola, FL: BioNews Services, LLC.
  12. "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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