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European Cystic Fibrosis Society’s (ECFS) Conference | 2024 | News | Updates | iPharmaCenter

Vertex Shares New Insights at European Cystic Fibrosis Conference Highlighting Major Advantages of TRIKAFTA Treatment

Vertex Pharmaceuticals revealed promising data at the 47th European Cystic Fibrosis Society’s (ECFS) Conference, which took place from June 5-8, 2024, in Glasgow, Scotland. The conference showcased significant outcomes of TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor), also branded as KAFTRIO in the EU and the UK, in treating cystic fibrosis (CF) patients with rare, non-F508del CFTR mutations.

Key Findings from the Clinical Study

A randomized, double-blind, Phase 3 study presented notable benefits of TRIKAFTA for CF patients with rare mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

The treatment group exhibited a 9.2 percentage point improvement in lung function, as assessed by ppFEV1, and a significant enhancement in CFTR function, indicated by a mean sweat chloride concentration reduction of 28.3 mmol/L. Furthermore, there was a 72% annual reduction in pulmonary exacerbations.

The safety and tolerability of TRIKAFTA were consistent with its established safety profile.

Real-World Evidence from the Registry-Based Study

Vertex also disclosed interim analysis (IA) findings from an ongoing five-year registry-based study (abstract WS01.04), examining real-world data from CF patients in the U.S. (2019-20) and Germany (2020-21) who initiated treatment with TRIKAFTA® or KAFTRIO®. This study, the largest of its kind, includes data from over 16,000 CF patients in the U.S. Cystic Fibrosis Foundation Patient Registry (CFFPR) and approximately 3,000 patients in the German CF Registry.

The IA results underscored substantial, disease-modifying advantages of TRIKAFTA/KAFTRIO, showing a 76% reduction in the cumulative annual rate of pulmonary exacerbations in the U.S. and a 70% reduction in Germany compared to the pre-treatment year. Additionally, there was a 62% decrease in the death rate in the U.S. and an 84% decrease in Germany, alongside an 86% reduction in lung transplant rates in the U.S. and a 96% reduction in Germany, relative to the 2019 data from the respective registries. No new safety issues were identified.


These findings from both the clinical and real-world studies affirm the significant clinical and functional benefits of TRIKAFTA/KAFTRIO for CF patients, particularly those with rare CFTR mutations. The ongoing research continues to demonstrate the transformative impact of this treatment on long-term health outcomes for individuals with cystic fibrosis.


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