Summary

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• Symdeko (tezacaftor and ivacaftor) is indicated for the treatment of patients with cystic fibrosis (CF) age 6 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence.
• This summary is based on the review of two systematic review(s)/meta-analysis(es). ^[1-2]
• Monotherapy: No evidence of mortality or clinically relevant quality of life (QoL) improvements; insufficient evidence on lung function effects; no significant differences in adverse effects (AEs) compared to placebo.
• Dual Therapy: Improved QoL scores in the respiratory domain and FEV₁ % predicted compared to placebo at six months; reduced pulmonary exacerbation rates with lumacaftor 600 mg (HR 0.70, 95% CI 0.57 to 0.87) and lumacaftor 400 mg (HR 0.61, 95% CI 0.49 to 0.76).
• Triple Therapy: Greater relative and absolute changes in FEV₁ % predicted, with F508del/MF participants on elexacaftor-tezacaftor-ivacaftor showing a mean difference of 14.30 (95% CI 12.76 to 15.84); reduced pulmonary exacerbation rates in F508del/F508del participants at four weeks (OR 0.17, 99% CI 0.06 to 0.45) and 24 weeks (OR 0.29, 95% CI 0.14 to 0.60).
• Dual therapy with lumacaftor-ivacaftor was associated with early transient breathlessness (OR 2.05, 99% CI 1.10 to 3.83) and long-term increases in systolic (5.1 mmHg) and diastolic (4.1 mmHg) blood pressure over 120 weeks; these adverse effects were not reported with tezacaftor-ivacaftor.
• Triple therapy showed no deaths and little to no difference in adverse events compared to control, indicating a comparable or better safety profile relative to dual therapies.
• There is no population types or subgroups information available in the reviewed studies.