Vertex Reports ALYFTREK Data on Pancreatic Function Recovery in Young CF Patients
Vertex Pharmaceuticals presented interim results from an open-label extension study of ALYFTREK in children aged 2 to 5 with cystic fibrosis. Some participants regained exocrine pancreatic function and could stop pancreatic enzyme replacement therapy. The data were shared at the North American Cystic Fibrosis Conference.
Vertex's interim findings come from Study 106 Cohort 2, an ongoing open-label extension trial. Of 48 children aged 2 to 5 eligible to stop pancreatic enzyme replacement therapy, 18 discontinued it for a mean of about 8.6 weeks. Across all 66 enrolled children, half were off PERT at the data cutoff. Mean fecal elastase-1 rose 110.2 µg/g to 239.7 µg/g, with 48.6% reaching sufficiency.
Exocrine pancreatic insufficiency affects roughly 90% of children with CF within their first year and was long viewed as permanent, requiring lifelong enzyme treatment and driving other complications. ALYFTREK's use in this age group remains investigational. Vertex also presented abstracts on CFTR modulators, including one on reduced IV antibiotic use in people 12 and older.
If these interim results hold, families of young children with cystic fibrosis could face fewer daily treatment burdens, since pancreatic enzyme therapy shapes mealtimes and routines. Clinicians may gain reason to consider earlier intervention, though longer follow-up is needed to confirm durability. Payers and health systems could see shifting costs. The findings cover a narrow age group and remain investigational, so implications for older patients are uncertain.