Vertex trial shows Alyftrek restores pancreatic function

Vertex Pharmaceuticals presented new interim data on October 9, 2026, from an open-label extension study of Alyftrek (vanzacaftor/tezacaftor/deutivacaftor) in children ages 2 to 5 with cystic fibrosis. The findings, unveiled at the North American Cystic Fibrosis Conference in Atlanta, Georgia, showed restored exocrine pancreatic function, previously thought to be irreversible.

In the Study 106 Cohort 2 substudy evaluating 48 eligible children after at least 48 weeks of treatment, mean fecal elastase-1 reached 239.7 μg/g, an average increase of 110.2 µg/g from baseline. Nearly half of the children, or 48.6%, reached the pancreatic sufficiency threshold of 200 µg/g or higher.

The trial demonstrated that 18 of the 48 eligible children (37.5%) successfully discontinued pancreatic enzyme replacement therapy (PERT) for a mean duration of 8.6 weeks, with a range of 1.9 to 17.6 weeks. Another 7 participants had stopped PERT before substudy eligibility. Across the entire enrolled cohort of 66 children, 50% were not taking PERT at the time of the data cut.

Carmen Bozic, Executive Vice President of Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex, stated that the Phase 3 open-label findings demonstrate that pancreatic organ failure can in fact be reversed. The use of Alyftrek in patients aged 2 to 5 remains investigational.