Vertex to Present New Data on ALYFTREK® at the North American Cystic Fibrosis Conference
Business Wire•09/10/2026•13:15 ET
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Key Highlights
- ➤48 children treated at least 48 weeks; mean FE-1 rose 110.2 µg/g
- ➤48.6% of children reached pancreatic sufficiency threshold of ≥200 µg/g
- ➤18 of 48 eligible children discontinued PERT for mean 8.6 weeks
- ➤50% of 66 enrolled children were not on PERT at data cut
- ➤ALYFTREK use in children ages 2 to 5 remains investigational
Expert Statements
Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex
“The data presented at NACFC underscore the safety and efficacy of our CF medicines in the real world and in clinical trials in younger age groups. The data from the Phase 3 open-label study in 2 to 5 year olds evaluating exocrine pancreatic function represent a profound shift in how we understand the benefits of treating cystic fibrosis in its earliest stages”
Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex
“For decades, pancreatic organ failure and resulting exocrine pancreatic insufficiency has been accepted as an irreversible consequence of CF, but these remarkable data in younger children treated with ALYFTREK show that, in fact, pancreatic failure can be reversed.”
Vertex to Present New Data on ALYFTREK(®) at the North American Cystic Fibrosis Conference
- Data on ALYFTREK in children ages 2 to 5 years demonstrate recovery of pancreatic function in some children –
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