On 18 May at the 2025 American Thoracic Society (ATS) International Conference, a poster presentation by Sivagurunathan Sutharsan and colleagues from University Medicine Essen, Ruhrlandklinik, provided compelling long-term efficacy data for elexacaftor/tezacaftor/ivacaftor (ETI) therapy in cystic fibrosis (CF) patients. The research demonstrated significant improvements across multiple clinical parameters over the extended follow-up period.
The 42-month observational cohort study included 85 patients with cystic fibrosis (pwCF) possessing at least one F508del allele, with a mean baseline age of 37±12 years (53.5% male). Inspiratory vital capacity increased by 8% (95% confidence interval [CI] 2.5–9.2, p=0.361) while pulmonary exacerbations decreased dramatically by 56.7% (p<0.0001). Quality of life, as measured by the Cystic Fibrosis Questionnaire-Revised score, improved by 26.3 points (95% CI 19.9–36.0, p<0.0001), reflecting meaningful patient-centred benefits. Notable laboratory findings included a significant decrease in sweat chloride concentration by 58.5mmol/g (p<0.0001) and haemoglobin A1c reduction by 2.25% (p<0.0001). Liver function parameters showed total bilirubin increased by 0.1mmol/L (p < 0.05), as well as improvement with glutamic-oxaloacetic transaminase decreasing by 8.5U/L and glutamate pyruvate alanine aminotransferase decreasing by 8.0U/L (both p<0.0001). The frequency of Pseudomonas aeruginosa detection decreased by 32.2% (p<0.0001), addressing a significant challenge in CF management.
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These findings reinforce ETI therapy as the gold standard therapy in the CF treatment landscape. Since its Food and Drug Administration (FDA) approval in 2019 and subsequent EMA approval in 2020, Vertex Pharmaceuticals’ Trikafta (ETI and ivacaftor) has revolutionised CF care, with global sales reaching $10.2bn in 2024, reflecting an 89.4% compound annual growth rate between 2019 and 2024. Key opinion leaders previously interviewed by leading data and analytics company GlobalData stated that “Trikafta appears to be the most effective choice in those people eligible for it. So, that’s the one that we use the most if a patient can use it.” KOLs also highlighted Trifakta as a “lifelong medicine for patients unless something better comes around” in interviews conducted prior to the launch of next-generation cystic fibrosis transmembrane conductance regulator (CFTR).
While the study results support the continued use of ETI in the F508del allele homozygous or heterozygous population following Vertex’s December 2024 FDA approval, which expanded Trikafta’s label to include 94 additional non-F508del CFTR mutations, similar real-world studies will need to be conducted to support its use in this broader population to achieve its full commercial potential. The published long-term data suggests that Trikafta could maintain its dominant position in the CF market in the mid-term while Vertex continues its efforts to develop and commercialise next-generation therapies such as Alyftrek, which was approved in the US in 2024 and the UK in 2025, as well as receiving a positive opinion from Committee for Medicinal Products for Human Use in April 2025, in order to reinforce Vertex as the leader in CF globally.
The real-world results presented by Sutharsan and colleagues substantiate Trifakta as an effective long-term therapy for pwCF with at least one F508del allele, providing new insights into laboratory markers while reinforcing its therapeutic value across various clinical outcomes in this patient population.
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