Ivacaftor

(Kalydeco®)

Kalydeco®

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Drug updated on 4/17/2024

Dosage FormTablet (oral; 150 mg); Granule (oral; 5.8 mg, 13.4 mg, 25 mg, 50 mg, 75 mg)
Drug ClassCFTR potentiators
Ongoing and
Completed Studies
ClinicalTrials.gov

Indication

  • Indicated for the treatment of cystic fibrosis (CF) in patients age 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data. If the patient’s genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use.

Summary
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  • Ivacaftor (Kalydeco) is indicated for the treatment of cystic fibrosis in patients aged 1 month and older who have at least one mutation in the CFTR gene that responds to ivacaftor, based on clinical or in vitro assay data.
  • Fifteen studies were reviewed, which provided comprehensive insights into ivacaftor's clinical performance within the broader CFTR modulator landscape.
  • As part of triple therapy with elexacaftor and tezacaftor, ivacaftor demonstrates substantial improvements in lung function, quality of life, BMI across several studies, and shows a reduction in acute pulmonary exacerbations, signifying its potent role as a therapeutic regimen for cystic fibrosis.
  • Dual therapy combinations such as lumacaftor-ivacaftor and tezacaftor-ivacaftor show meaningful improvements but with variations in safety profiles. The combination lumacaftor-ivacaftor was associated with early transient breathlessness and long-term increases in blood pressure, concerns not highlighted with tezacaftor-ivacaftor.
  • Real-world evidence suggests that ivacaftor, alone or combined, is generally well-tolerated by patients, aligning adverse events observed during clinical trials, making it safe compared to other drugs used for treating cystic fibrosis.
  • Efficacy varies across different genetic mutations, age groups, and the presence of specific comorbidities like Cystic Fibrosis Related Diabetes (CFRD). Significant benefits were noted, particularly in populations with one or two copies of the F508del mutation when treated with triple therapy, showing a favorable safety profile across various mutations.
  • The impact on the pediatric population, especially those under years of age, is promising with notable improvements in lung function and growth parameters without significant safety concerns, highlighting the potential for early intervention in suitable candidates.
  • Ivacaftor marks a significant leap in care by addressing the underlying genetic cause of the disease rather than just managing symptoms. These findings suggest a reevaluation of dual therapies in favor of triple therapy due to superior efficacy and manageable safety profiles, however, age restrictions and the precise genotypic profile of the patient are critical in therapy selection.

Product Monograph / Prescribing Information

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

Systematic Reviews / Meta-Analyses

Document TitleYearSource
Use of elexacaftor+tezacaftor+ivacaftor in individuals with cystic fibrosis and at least one F508del allele: a systematic review and meta-analysis.2023Jornal Brasileiro de Pneumologia
Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del).2023The Cochrane Database of Systematic Reviews
Efficacy and safety profile of elexacaftor-tezacaftor-ivacaftor triple therapy on cystic fibrosis: a systematic review and single arm meta-analysis.2023Frontiers in Pharmacology
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.2022The Cochrane 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
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).2020The Cochrane Database of Systematic Reviews
A systematic review of the clinical efficacy and safety of CFTR modulators in cystic fibrosis.2019Scientific Reports
Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis (review).2019The Cochrane Database of Systematic Reviews

Clinical Practice Guidelines