Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) has announced promising new data showcasing the clinical and quality-of-life benefits of CFTR modulators, particularly ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor), during the 48th European Cystic Fibrosis Conference held June 4–7, 2025, in Milan, Italy. ALYFTREK, currently approved in the U.S. and U.K., is under regulatory review in the EU, Canada, Australia, New Zealand, and Switzerland.
Key data presented at the conference included a pooled analysis of CFTR modulators, which reinforced the correlation between reductions in sweat chloride (SwCl)—a biomarker of improved CFTR function—and better clinical outcomes in cystic fibrosis (CF) patients. Notably, individuals with SwCl levels under 60 mmol/L experienced more stable lung function, fewer pulmonary flare-ups, enhanced nutritional status, and improved overall quality of life. The most significant numerical benefits were observed in patients with SwCl levels under 30 mmol/L.
In addition, Vertex shared results from a post hoc analysis of Phase 3 trials (abstract WS19.04), which showed that ALYFTREK significantly improved health-related quality of life in adolescents and adults, and enhanced CF symptom management and functional ability in children aged 6 to 11 years—surpassing outcomes seen with TRIKAFTA.
“These findings reinforce the critical role of sweat chloride reduction in driving better CFTR function and long-term clinical benefits,” said Professor Isabelle Fajac, pulmonologist and Professor of Physiology at APHP-Université Paris Cité. “What’s particularly compelling is ALYFTREK’s potential to provide even greater improvements in quality of life and health outcomes compared to earlier CFTR modulators like TRIKAFTA.”