Therapy Areas: Hereditary Disorders
Vertex Presents Data Demonstrating Significant Benefits of Long-Term Treatment with CFTR Modulators at the European Cystic Fibrosis Conference
12 June 2023 - - US-based biotechnology company Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) said that 12 scientific abstracts on the company's portfolio of cystic fibrosis medicines were presented at this year's European Cystic Fibrosis Society's 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 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 UK 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 US and 83% in Germany overall compared to pre-TRIKAFTA baseline.

The rate of death was 72% lower in the US and 82% lower in Germany, the rate of lung transplant was 85% lower in the US and 100% lower in Germany, compared to 2019 (pre-TRIKAFTA) US 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.

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.

The company has multiple approved medicines that treat the underlying cause of cystic fibrosis -- a rare, life-threatening genetic disease -- and has several ongoing clinical and research programs in CF.

Beyond CF, Vertex has a robust clinical pipeline of investigational small molecule, mRNA, cell and genetic therapies (including gene editing) in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, acute and neuropathic pain, type 1 diabetes and alpha-1 antitrypsin deficiency.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London.


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