Press release
Vertex to Present Data Demonstrating Significant Benefits of Long-Term and Early Treatment With CFTR Modulators at the European Cystic Fibrosis Conference
- TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) real-world safety and effectiveness interim results show improved lung function and significant
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n- TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) real-world safety and effectiveness interim results show improved lung function and significant reductions in risk of pulmonary exacerbations, lung transplant and death for people with cystic fibrosis (CF) -\n\n- Study in people with CF (F/F or F/MF genotypes) treated with TRIKAFTA® shows no mean loss of lung function after two years compared to those not treated with a CFTR modulator -\n\n- Long-term real-world study results show significant benefits of initiating KALYDECO® (ivacaftor) at young age -\n\n BOSTON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that five scientific abstracts on the company’s portfolio of cystic fibrosis (CF) medicines will be presented at the European Cystic Fibrosis Society's (ECFS) 45th European Cystic Fibrosis Conference held June 8-11, 2022, in Rotterdam, the Netherlands.\n\nVertex will present the first analysis of data collected in the U.S. CF Foundation Patient Registry (CFFPR) of over 16,000 people with CF treated with TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for an average of nine months. This first interim analysis of an ongoing five-year post-authorization study (abstract WS22.05) showed that real-world treatment with TRIKAFTA® was associated with improved lung function and a 77% reduced risk of pulmonary exacerbations compared to pre-TRIKAFTA® baseline, as well as an 87% lower risk of lung transplant and a 74% lower risk of death, compared to the historical 2019 U.S. CFFPR population. No new safety concerns were identified.\n\nVertex will also present data comparing the annual rate of lung function change in people with CF ages 12 years and older with two F508del mutations (F/F) or one F508del mutation and one minimal function mutation (F/MF) treated with TRIKAFTA® in pivotal studies and an open-label extension study compared to propensity-score matched historical CFTR-modulator-untreated controls from the U.S. CFFPR (abstract WS22.04). Results show that TRIKAFTA® demonstrated on average no decrease in ppFEV1 over a two-year period in this population, in contrast to declines seen in the matched controls. The analysis indicates that treatment with TRIKAFTA® has a significant impact on the trajectory of CF lung disease.\n\nAdditionally, Vertex will present data from a l...