Vertex Presents Data Demonstrating Significant Benefits of Long-Term Treatment With CFTR Modulators at th

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2023-06-09 15:00:00  • 21 mins

-Interim results of largest real-world study of TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) showed sustained improvement in lung function, reduction in pulmonary exacerbations frequency and lower rates of lung transplant and death for people with cystic fibrosis-

- Twelve presentations add to the body of evidence supporting the use of CFTR modulators for all eligible people with cystic fibrosis-

Vertex Pharmaceuticals Incorporated VRTX today announced that 12 scientific abstracts on the company's portfolio of cystic fibrosis (CF) medicines were presented at this year's European Cystic Fibrosis Society's (ECFS) 46th European Cystic Fibrosis Conference held June 7-10, 2023, in Vienna, Austria. Together, the data presented show the long-term benefits of treatment with CFTR modulators as well as the importance of treating the underlying cause of CF as early in life as possible. Key data presented at this year's conference are highlighted below.

Vertex presented an interim analysis (IA) of a registry-based study of real-world data collected from people with CF and treated with TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor), also known in the European Union and in the U.K. as KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor, including over 16,000 people with CF from the Cystic Fibrosis Foundation Patient Registry (CFFPR) and nearly 3,000 from the German CF Registry. This ongoing five-year post-authorization study (abstract WS16.03) is the largest real-world study of people with CF treated with TRIKAFTA to date. The IA showed clinically significant disease-modifying benefits for TRIKAFTA, including improved lung function and a 79% reduction of pulmonary exacerbations in the U.S. and 83% in Germany overall compared to pre-TRIKAFTA baseline. The rate of death was 72% lower in the U.S. and 82% lower in Germany, the rate of lung transplant was 85% lower in the U.S. and 100% lower in Germany, compared to 2019 (pre-TRIKAFTA) U.S. CFFPR and German CF Registry populations. No new safety concerns were identified.

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