ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor)

October 9, 2026: Vertex Pharmaceuticals (NASDAQ: VRTX) reported interim NACFC data for ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor) in an ongoing open-label extension study (VX22-121-106, Cohort 2) in children with cystic fibrosis ages 2 to 5, an investigational use. In a PERT discontinuation substudy of 48 children treated for at least 48 weeks, mean fecal elastase-1 was 239.7 μg/g, a mean increase of 110.2 μg/g from baseline, and 48.6% reached the pancreatic sufficiency threshold of at least 200 μg/g.

Among the 48 substudy-eligible children, 18 (37.5%) successfully discontinued pancreatic enzyme replacement therapy for a mean of 8.6 weeks (range 1.9-17.6 weeks) at interim analysis; 7 others had discontinued PERT before substudy eligibility. Across the full enrolled cohort of 66 children, 50% were not on PERT at data cut. Vertex said the findings suggest recovery of exocrine pancreatic function in some children.

The company also said additional NACFC abstracts covered: long-term post-hoc 96-week open-label extension data on VNZ/TEZ/D-IVA reducing IV antibiotic use in people with CF aged 12 and older; a natural history study of exocrine pancreatic function in infants under 12 months; and lifetime treatment burden and cost of PERT. These data were presented at NACFC, held October 7-10 in Atlanta.