Today, Vertex Pharmaceuticals announced the presentation of new data on TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), known in the European Union and the U.K. as KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor, at the 47th European Cystic Fibrosis Society (ECFS) Conference held June 5-8, 2024, in Glasgow, Scotland.
A Phase 3 randomized, double-blind study (abstract WS06.04) revealed significant clinical benefits for individuals with CF who have rare, non-F508del mutations in the CFTR gene. Compared to placebo, TRIKAFTA® improved lung function by 9.2 percentage points (ppFEV1), reduced sweat chloride concentrations by 28.3 mmol/L, and decreased pulmonary exacerbations by 72% annually. The safety profile remained consistent with previous findings for TRIKAFTA®.
Vertex also shared interim analysis from a registry-based study of real-world data from U.S. and German patients (abstract WS01.04). This ongoing five-year post-authorization study, the largest of its kind, involves over 16,000 patients from the U.S. Cystic Fibrosis Foundation Patient Registry and around 3,000 from the German CF Registry. The interim analysis showed that TRIKAFTA®/KAFTRIO® significantly reduced pulmonary exacerbations by 76% in the U.S. and 70% in Germany, decreased death rates by 62% and 84%, respectively, and lowered lung transplant rates by 86% in the U.S. and 96% in Germany. No new safety concerns were identified.
Dr. Carmen Bozic, Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex, remarked, “The breadth of TRIKAFTA data presented at ECFS underscores its significant potential as a disease-modifying treatment for CF, fundamentally altering the course of the disease and enhancing the lives of patients.”
Additional Vertex presentations at the conference included:
- Abstract WS15.02: Real-World Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in CF Patients with Non-F508del CFTR Genotypes
- Abstract EPS10.08: LONGITUDE Study on Long-term Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 6-11 Years
- Abstract P096: Qualitative Interviews on the Migration of the Preschool Pictorial CFQ-R
- Abstract EPS6.05: Clinical Outcomes in Concurrent Elexacaftor/Tezacaftor/Ivacaftor Treated vs. Ineligible Cohorts during COVID-19
- Abstract P063: Real-World Benefits of Elexacaftor/Tezacaftor/Ivacaftor in the TRAJECTORY Study
- Abstract P072: Impact of Elexacaftor/Tezacaftor/Ivacaftor in Italy from a CF Center Study
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting over 92,000 people globally. It impacts multiple organs, causing progressive lung damage due to thick, sticky mucus. CF results from defective CFTR proteins caused by specific gene mutations, with the most common being F508del. Treatments have improved life expectancy, with median age of death now in the 30s.
About TRIKAFTA®
TRIKAFTA® is an oral medication designed to enhance the quantity and function of CFTR proteins at the cell surface in patients with specific CFTR mutations. It is approved for use in patients aged 2 years and older with at least one F508del mutation or another responsive mutation.
Important Safety Information
Patients should discuss their medical history and all medications with their doctor before starting TRIKAFTA®. It may interact with certain medications and is not recommended during pregnancy or breastfeeding. Common side effects include liver enzyme elevation, respiratory infections, abdominal pain, and headaches. Serious side effects can include liver damage, allergic reactions, and cataracts in children. For detailed safety information, patients should consult the full U.S. Prescribing Information for TRIKAFTA®.
