Summary

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• Tezacaftor and ivacaftor (Symdeko) is indicated for the treatment of patients with cystic fibrosis aged 6 years and older who are homozygous for the F508del mutation or have at least one mutation in the CFTR gene that responds to this drug combination.
• Two systematic reviews/meta-analyses studies provided a comparative safety and effectiveness analysis between Symdeko and other CFTR modulators used in treating cystic fibrosis.
• The studies underscore that all reviewed CFTR modulators, including tezacaftor/ivacaftor, were generally well-tolerated. Compared to lumacaftor/ivacaftor (LUM/IVA), Symdeko showed a potentially better safety profile due to lower rates of respiratory-related adverse events and discontinuation.
• Regarding blood pressure impact, LUM/IVA was associated with increases over 120 weeks, while no such effect was reported for tezacaftor/ivacaftor. This suggests a superior safety profile for Symdeko concerning blood pressure effects.
• Both tezacaftor/ivacaftor (Symdeko) and LUM/IVA demonstrated similar small improvements in quality of life measures and respiratory function. However, reduction rates in pulmonary exacerbations observed when these therapies were added to ivacaftor indicate an additional benefit from using Symdeko specifically.
• Although there is a noted gap in safety data for younger children under 12 years old taking TEZ/IVA therapy like Symdeko, it has been particularly effective among patients homozygous for F508del mutations or those having at least one responsive mutation, underscoring its clinical value despite the limited options currently available.