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 –

- Additional abstracts on clinical and real-world evidence on CFTR modulators also presented -

BOSTON, October 09, 2026--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced new interim data from the ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor) 2 to 5 years old open-label extension study, highlighting recovery of exocrine pancreatic function in some children in that age group, allowing those children to discontinue pancreatic enzyme replacement therapy (PERT). Pancreatic exocrine insufficiency in children with cystic fibrosis (CF) was previously believed to be irreversible. These data were presented at the North American Cystic Fibrosis Conference (NACFC).

"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," said 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."

ALYFTREK interim data on exocrine pancreatic function

Presented as a late breaker poster at NACFC, the new interim data from 48 children who enrolled in a PERT discontinuation substudy VX22-121-106 (Study 106) Cohort 2, an ongoing open-label extension study of vanzacaftor/tezacaftor/deutivacaftor in children 2 to 5 years of age, demonstrate that early intervention with vanzacaftor/tezacaftor/deutivacaftor has a positive impact on exocrine pancreatic insufficiency (EPI) and can help achieve PERT independence. EPI is one of the earliest and most severe manifestations of CF, occurring in ~90% of children during the first year of life, requiring lifelong treatment, and driving serious downstream comorbidities. Specifically, data from the interim analysis conducted after children received at least 48 weeks of vanzacaftor/tezacaftor/deutivacaftor treatment showed:

• Meaningful improvement in fecal elastase-1 (FE-1) levels demonstrates restored pancreatic function: Mean FE-1, a well-established endpoint of exocrine pancreatic function, was 239.7 μg/g, representing a mean increase of 110.2 µg/g from baseline, with nearly half (48.6%) of children reaching the pancreatic sufficiency threshold of ≥200 µg/g.

• Of the 48 children eligible for the PERT discontinuation substudy, 18 (37.5%) successfully discontinued PERT for a mean duration of 8.6 weeks (range: 1.9 to 17.6 weeks) at the time of the interim data analysis. Additionally, 7 participants had discontinued PERT prior to the substudy eligibility. Across the full enrolled cohort of 66 children, 50% were not on PERT at the time of the data cut. The ability to discontinue PERT suggests reversal of prior pancreatic exocrine organ failure for those participants.