Milan– Vertex Pharmaceuticals has unveiled new clinical findings at the 48th European Cystic Fibrosis Conference (ECFS), underscoring the benefits of its CFTR modulator therapies, particularly ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor). The drug, approved in the U.S. and U.K. and under review in several other countries, is showing promising results in improving both clinical outcomes and quality of life for people with cystic fibrosis (CF).
One key presentation detailed a pooled analysis of CFTR modulators, including ALYFTREK, showing that reductions in sweat chloride levels — a biomarker for CFTR function — are strongly associated with better health outcomes. Patients with sweat chloride levels below 60 mmol/L experienced more stable lung function, fewer pulmonary exacerbations, improved nutrition, and enhanced quality of life. Those with levels under 30 mmol/L saw even greater benefits, though the differences were not statistically significant compared to the 30–60 mmol/L range.
Additional data from a post hoc analysis of Phase 3 randomized and open-label trials suggested that ALYFTREK led to improved health-related quality of life compared to TRIKAFTA®, especially in adolescents and adults. Children aged 6–11 treated with ALYFTREK also showed better symptom control and general functioning.
“These new data further demonstrate that reducing sweat chloride via treatment with CFTR modulators drives improved CFTR function and may ultimately result in better outcomes for patients,” said Professor Isabelle Fajac, a pulmonologist and professor at APHP-Université Paris Cité. “Importantly, the data also indicate that ALYFTREK… may drive improved quality of life and other health-related outcomes above and beyond even what we’ve seen with CFTR modulators to date.”
The findings add to growing evidence that next-generation CFTR therapies like ALYFTREK could represent a significant step forward in CF care.
