Vertex presents promising new data from cystic fibrosis trials
Ella Day | June 9, 2025 | News story | Medical Communications, Research and Development |Â Â European Cystic Fibrosis Conference, Rare Diseases, Vertex, clinical trial, cystic fibrosisÂ
Vertex Pharmaceuticals has presented new data showing the clinical and quality-of-life benefits of its cystic fibrosis (CF) therapies, particularly Alyftrek (deutivacaftor/tezacaftor/vanzacaftor). The findings, shared at the 48th European Cystic Fibrosis Conference, 4 to 7 June 2025, Milan, Italy, highlight the potential of CF transmembrane conductance regulator (CFTR) modulators to significantly improve patient outcomes.
A combined analysis of CFTR modulators found that greater restoration of CFTR function – measured by reduced sweat chloride levels (SwCl) – was linked to better health outcomes. Patients with SwCl levels below 60mmol/L showed more stable lung function, fewer pulmonary exacerbations, better nutritional status and improved quality of life.
Alyftrek in particular outperformed Trikafta (elexacaftor/tezacaftor/ivacaftor), also known as Kaftrio in the UK and EU, in improving symptoms, indicating a promising advance in CF care.
CF is a rare, life-shortening genetic disease affecting over 109,000 people worldwide. It results from a defective or missing CFTR protein, leading to thick, sticky mucus that causes persistent lung infections and progressive respiratory decline. CFTR modulators aim to correct or enhance the protein’s function to restore cellular balance.
Vertex remains the leading provider of CF treatments, with over 75,000 patients currently receiving its therapies, representing around two-thirds of those eligible for CFTR modulators.
Ella Day
9/6/25
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