Skip to main content

Elexacaftor/Ivacaftor/Tezacaftor: First Approval


A fixed-dose combination tablet of the cystic fibrosis transmembrane conductance regulator (CFTR) corrector tezacaftor and the CFTR potentiator ivacaftor with the next-generation CFTR corrector elexacaftor (hereafter referred to as elexacaftor/ivacaftor/tezacaftor) [Trikafta™] has been developed by Vertex Pharmaceuticals Inc. to treat patients with the most common cystic fibrosis mutation (F508del). Its use has been associated with statistically significant and/or clinically meaningful improvements in lung function and respiratory-related quality of life compared with comparator regimens (placebo or ivacaftor/tezacaftor) in multinational phase II and III studies, and in October 2019 elexacaftor/ivacaftor/tezacaftor was approved by the US FDA for the treatment of cystic fibrosis in patients aged ≥ 12 years who have ≥ 1 F508del mutation in the CFTR gene. A regulatory assessment for elexacaftor/ivacaftor/tezacaftor as a treatment for cystic fibrosis is underway in the EU. This article summarizes the milestones in the development of elexacaftor/ivacaftor/tezacaftor leading to this first approval for the treatment of cystic fibrosis in patients aged ≥ 12 years who have ≥ 1 F508del mutation in the CFTR gene.

This is a preview of subscription content, access via your institution.


  1. Gentzsch M, Mall MA. Ion channel modulators in cystic fibrosis. Chest. 2018;154(2):383–93.

    Article  Google Scholar 

  2. US FDA. FDA approves new breakthrough therapy for cystic fibrosis. 2019. Accessed 23 Oct 2019.

  3. Ren CL, Morgan RL, Oermann C, et al. Cystic Fibrosis Foundation pulmonary guidelines: use of cystic fibrosis transmembrane conductance regulator modulator therapy in patients with cystic fibrosis. Ann Am Thorac Soc. 2018;15(3):271–80.

    Article  Google Scholar 

  4. Rowe SM, Taylor-Cousar JL. Advancing CFTR modulator therapy for the vast majority of patients with cystic fibrosis [abstract no. S15.2]. Pediatr Pulmonol. 2019;54(Suppl 2):108–9.

    Google Scholar 

  5. Middleon PG, Mall MA, Dřevínek LC, et al. Elexacaftor–tezacaftor–ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med. 2019;381:1809–19.

    Article  Google Scholar 

  6. Vertex Pharmaceuticals Inc. Trikafta™ (elexacaftor, tezacaftor and ivacaftor tablets; ivacaftor tablets): US prescribing information. 2019. Accessed 23 Oct 2019.

  7. Vertex Pharmaceuticals Inc. Vertex announces European Medicines Agency Marketing Authorization Application validation for VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination treatment in cystic fibrosis. 2019. Accessed 4 Nov 2019.

  8. Vertex Pharmaceuticals Inc. Orkambi® (lumacaftor/ivacaftor) tablets, for oral use: US prescribing information. 2018. Accessed 4 Nov 2019.

  9. Keating D, Marigowda G, Burr L, et al. VX-445-tezacaftor-ivacaftor in patients with cystic fibrosis and one or two Phe508del alleles. N Engl J Med. 2018;379(17):1612–20.

    CAS  Article  Google Scholar 

  10. Heijerman HGM, McKone EF, Downey DG, et al. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial. Lancet. 2019.

    Article  PubMed  Google Scholar 

Download references

Author information

Authors and Affiliations


Corresponding author

Correspondence to Sheridan M. Hoy.

Ethics declarations


The preparation of this review was not supported by any external funding.

Conflict of interest

During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the author on the basis of scientific completeness and accuracy. Sheridan Hoy is a salaried employee of Adis International Ltd/Springer Nature, is responsible for the article content and declares no relevant conflicts of interest.

Additional information

Enhanced material for this AdisInsight Report can be found at

This profile has been extracted and modified from the AdisInsight database. AdisInsight tracks drug development worldwide through the entire development process, from discovery, through pre-clinical and clinical studies to market launch and beyond.

Rights and permissions

Reprints and Permissions

About this article

Verify currency and authenticity via CrossMark

Cite this article

Hoy, S.M. Elexacaftor/Ivacaftor/Tezacaftor: First Approval. Drugs 79, 2001–2007 (2019).

Download citation

  • Published:

  • Issue Date:

  • DOI: