Drug updated on 11/9/2023

Dosage FormTablet (oral; 150 mg); Granule (oral; 25 mg, 50 mg, 75 mg)
Drug ClassCystic fibrosis transmembrane conductance regulator (CFTR) potentiators
Ongoing and
Completed Studies


  • For the treatment of cystic fibrosis (CF) in patients age 4 months and older who have one mutation in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data.

Product Monograph / Prescribing Information

Document TitleYearSource
Kalydeco (ivacaftor) Prescribing Information.2023Vertex Pharmaceuticals Incorporated, Boston, MA

Systematic Reviews / Meta-Analyses

Document TitleYearSource
Efficacy and Safety of Elexacaftor-Tezacaftor-Ivacaftor in the Treatment of Cystic Fibrosis: A Systematic Review. 2023Children
Cystic Fibrosis Transmembrane Conductance Regulator Protein Modulators in Children and Adolescents with different CF Genotypes - Systematic Review and Meta-Analysis. 2023Current Reviews in Clinical and Experimental Pharmacology
The Effects of Elexacaftor, Tezacaftor, and Ivacaftor (ETI) on Blood Glucose in Patients With Cystic Fibrosis: A Systematic Review. 2023Cureus
Medical interventions for chronic rhinosinusitis in cystic fibrosis.2022Cochrane Database of Systematic Reviews
Newly discovered cutting-edge triple combination cystic fibrosis therapy: a systematic review.2022Cureus
Effect of CFTR modulators on anthropometric parameters in Individuals with cystic fibrosis: an evidence analysis center systematic review.2021Journal of the Academy of Nutrition and Dietetics
Real-world safety of CFTR modulators in the treatment of cystic fibrosis: a systematic review.2021Journal of Clinical Medicine
The effectiveness and value of novel treatments for cystic fibrosis. 2021Journal of Managed Care & Specialty Pharmacy
Real-World Outcomes of Ivacaftor Treatment in People with Cystic Fibrosis: A Systematic Review. 2021Journal of Clinical Medicine
Modulator treatments for cystic fibrosis: effectiveness and value.2020ICER
From ivacaftor to triple combination: a systematic review of efficacy and safety of CFTR modulators in people with cystic fibrosis.2020International Journal of Molecular Sciences
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).2020Cochrane Database of Systematic Reviews
Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis (review).2019Cochrane Database of Systematic Reviews
A systematic review of the clinical efficacy and safety of CFTR modulators in cystic fibrosis.2019Scientific Reports
Efficacy and Safety of CFTR Corrector and Potentiator Combination Therapy in Patients with Cystic Fibrosis for the F508del-CFTR Homozygous Mutation: A Systematic Review and Meta-analysis. 2018Advances in Therapy
Correctors (specific therapies for class II CFTR mutations) for cystic 2018Cochrane Database of Systematic Reviews

Clinical Practice Guidelines

Document TitleYearSource
ECFS best practice guidelines: the 2018 revision.2018Journal of Cystic Fibrosis