Vertex Presents New Data on ALYFTREK® at European Cystic Fibrosis Conference

About this update from Vertex Pharmaceuticals Incorporated

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data demonstrating the potentially transformative impact of treating cystic fibrosis (CF) with ALYFTREK ® (vanzacaftor/tezacaftor/deutivacaftor) 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 (F/F) and those who have F508del/minimal function mutations (F/MF) on ALYFTREK had further improvement in CFTR function from a TRIKAFTA ® baseline as measured by sweat chloride (SwCl), with 65% having achieved SwCl <30 mmol/L after treatment with ALYFTREK. Vertex also presented Phase 3 data of children ages 1 to <2 with TRIKAFTA (elexacaftor/tezacaftor/ivacaftor). 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 <2. “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, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “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 <30mmol/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.” “As someone who has been treating people with CF for more than 20 years and whose center is involved in the ALYFTREK 2 to 5 years clinical program, I have seen firsthand how the medicine can help patients achieve better CFTR function through reduction in sweat chloride and improve other important markers of disease like exocrine pancreatic function,” said Professor Marcus A. Mall, M.D., Professor and Chair of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center at Charité Unive...

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