Vertex's TRIKAFTA® Shows Significant Clinical Benefits in Cystic Fibrosis Patients with Rare Mutations

10 June 2024 | Monday | News

Phase 3 study reveals improved lung function and reduced pulmonary exacerbations; real-world data confirm sustained benefits over three years, lowering lung transplant and death rates.
Image Source : Public Domain

Image Source : Public Domain

Results from a randomized, placebo-controlled study of TRIKAFTA® in people with cystic fibrosis with rare, non-F508del CFTR mutations showed statistically significant and clinically meaningful improvements in the primary and all secondary endpoints -

Interim results of largest real-world study of TRIKAFTA® showed sustained improvement in lung function at three years as well as lower rates of lung transplant and death in people with cystic fibrosis, compared to pre-TRIKAFTA® initiation -


Vertex Pharmaceuticals Incorporated  announced that data on 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, were presented at this year’s European Cystic Fibrosis Society’s (ECFS) 47th European Cystic Fibrosis Conference held June 5-8, 2024, in Glasgow, Scotland.

Data from a randomized, double-blind, Phase 3 study (abstract WS06.04) demonstrated that people with CF who have rare, non-F508del mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene responsive to TRIKAFTA® in vitro demonstrated clinical benefit from receiving TRIKAFTA®. Compared to placebo, lung function improved by 9.2 percentage points as measured by ppFEV1, CFTR function improved (as measured by mean sweat chloride concentration reductions of 28.3 mmol/L), and pulmonary exacerbations were reduced by 72% per year. Safety and tolerability were generally consistent with the established safety profile of TRIKAFTA®.

Vertex also presented the interim analysis (IA) of a registry-based study of real-world data collected from people with CF initiating TRIKAFTA® from 2019-20 in the U.S. and KAFTRIO® plus ivacaftor from 2020-21 in Germany (abstract WS01.04). The ongoing five-year post-authorization study is the largest real-world study of people with CF treated with TRIKAFTA®/KAFTRIO® to date, including more than 16,000 people with CF from the U.S. Cystic Fibrosis Foundation Patient Registry (CFFPR) and approximately 3,000 people with CF from the German CF Registry. The IA showed clinically meaningful, disease-modifying benefits for TRIKAFTA®/KAFTRIO®, including a 76% and 70% reduction in the cumulative annual rate of pulmonary exacerbations in the U.S. and in Germany, respectively, compared to the year prior to TRIKAFTA®/KATRIO® treatment. In addition, there was a 62% lower rate of death in the U.S. and 84% lower in Germany; and an 86% lower rate of lung transplant in the U.S. and 96% lower in Germany compared to the 2019 U.S. CFFPR and German CF Registry populations (pre-TRIKAFTA®/KAFTRIO®). No new safety concerns were identified.

“The breadth of TRIKAFTA data presented at ECFS is further evidence of the significant potential of this disease-modifying medicine,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “These studies demonstrate that TRIKAFTA is changing the course of CF treatment and the lives of those living with CF.”


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