-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-
BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: 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.
Vertex also presented final results of the nearly four-year TRIKAFTA® open-label follow-up study of the Phase 3 pivotal trials in people with CF ages 12 years and older with at least one F508del mutation in the CFTR gene (Late Breaking Science; Workshop 15). The results of this study are unprecedented, showing for the first time that treatment with TRIKAFTA® resulted in no decline in lung function over a four-year period.
“CFTR modulators markedly improve clinical outcomes of people living with CF as demonstrated in the large and growing quantity of data presented at ECFS this year,” said Professor Isabelle Fajac, Professor of Physiology, Cochin Hospital, Assistance Publique-Hôpitaux de Paris, Université Paris Cité, Paris, France. “The data on TRIKAFTA in particular demonstrate that this medicine improves lung function sustainably and in a real-world setting. It also reduces the risks of pulmonary exacerbation, death and lung transplant, and it is generally well tolerated.”
Additional Presentations
Other Vertex presentations at the conference this year include:
- Abstract EPS6.05 entitled “A Phase 3b study of the effects of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on cough and physical activity in people with cystic fibrosis (CF)”
- Abstract WS05.04 entitled “Safety and efficacy of ivacaftor (IVA) in children aged 1 to