Vertex Pharmaceuticals Shows ALYFTREK's Potential Impact in Children Aged 2 to 5

Vertex Pharmaceuticals announced data demonstrating the potentially transformative impact of treating cystic fibrosis with ALYFTREK in children ages 2 to 5, as well as data from 96-week interim analyses of two open-label extension studies of ALYFTREK in children 6 to 11 years and people 12 years and older demonstrating the long-term safety and efficacy profile of the medicine. The data, presented at the European Cystic Fibrosis Conference, show children ages 2 to 5 with vanzacaftor/tezacaftor/deutivacaftor-responsive genotypes including those who are homozygous for the F508del mutation and those who have F508del/minimal function mutations on ALYFTREK had further improvement in CFTR function from a TRIKAFTA baseline as measured by sweat chloride, with 65% having achieved SwCl less than30 mmol/L after treatment with ALYFTREK. Vertex also presented Phase 3 data of children ages 1 to less than2 with TRIKAFTA. Vertex plans to submit for global regulatory approvals of ALYFTREK in children ages 2 to 5 in the first half of 2026, and the company has begun global regulatory submissions for TRIKAFTA in children ages 1 to less than2. "The data we're presenting today bring us to the cusp of our 25-year mission to advance medicines that restore CFTR function to people living with CF," said Carmen Bozic, CMO. "They show that ALYFTREK is the first medicine to bring the majority of children ages 2 to 11 to SwCl below 30 mmol/L, which is incredibly exciting because SwCl less than30mmol/L is the median value seen in carriers who are known to have normal health and is a key marker of restoration of CFTR function." Data presented in children ages 2-5 treated with ALYFTREK: "A Phase 3 open-label clinical trial of vanzacaftor/tezacaftor/deutivacaftor in children aged 2-5 years with cystic fibrosis" was presented as a late-breaking abstract and oral presentation in the "Late-Breaking Science" session on June 5 from 5:00 p.m. to 6:30 p.m. GMT+1. Data from 67 children who all completed the 24-week, Phase 3, open-label study show that ALYFTREK was generally safe and well tolerated, consistent with the established safety profile. The primary endpoint of the study was safety and tolerability. Treatment with ALYFTREK resulted in a rapid, clinically meaningful improvement in CFTR function with a mean reduction in sweat chloride from a baseline on TRIKAFTA of -9.6 mmol/L through Week 24, with 92% of children achieving SwCl concentrations of less than60 mmol/L, and 65% of children reaching SwCl values of less than30 mmol/L. These improvements in CFTR function surpass those seen in trials with any other CFTR modulator in this age group. Data presented in children ages 1 to less than2 treated with TRIKAFTA: "A Phase 3, 24-Week, Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children with Cystic Fibrosis 12 to less than24 Months of Age" was featured in an oral presentation as part of the symposium "Clinical and functional impact of highly effective modulators" on June 4 from 3:00-4:30 p.m. GMT+1 and the abstract will be published in the Journal of Cystic Fibrosis:. Results from a 24-week, Phase 3, open-label study of TRIKAFTA in 54 enrolled children aged 12 to less than24 months was presented. The primary endpoint was safety and tolerability. TRIKAFTA was generally safe and well tolerated; the safety data are consistent with the established safety profile. Treatment with TRIKAFTA in this age group resulted in rapid, statistically significant and clinically meaningful decrease in SwCl, with a mean reduction of -71.8 mmol/L from a baseline without CFTR modulator treatment through Week 24, with 98.0% of children achieving concentrations less than60 mmol/L and 68.6% reaching less than30 mmol/L. The uses of ALYFTREK in children with CF 2 to 5 years old, and TRIKAFTA in children with CF 1 to less than2 years old, are investigational.