Press release

Vertex Highlights First Oral Presentation of Phase 3 Clinical Data of the Vanza Triple and New Data on Long-Term Impact of TRIKAFTA® at the North American Cystic Fibrosis Conference

– Phase 3 data on investigational vanza triple demonstrates non-inferiority to TRIKAFTA® in ppFEV1 and further improvement of CFTR function as measured by

articleVertex Pharmaceuticals IncorporatedSeptember 26, 20245/news/vertex-highlights-first-oral-presentation-phase-3-clinical-data-vanza-triple-and-new
Vertex Highlights First Oral Presentation of Phase 3 Clinical Data of the Vanza Triple and New Data on Long-Term Impact of TRIKAFTA® at the North American Cystic Fibrosis Conference

About this update from Vertex Pharmaceuticals Incorporated

– Phase 3 data on investigational vanza triple demonstrates non-inferiority to TRIKAFTA® in ppFEV1 and further improvement of CFTR function as measured by sweat chloride – – Real-world evidence and clinical studies of TRIKAFTA® continue to show sustained long-term benefits including improvement in pancreatic function in young patients – BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the first accepted medical presentations of the Phase 3 data on the investigational once daily vanzacaftor/tezacaftor/deutivacaftor (“vanza triple”) — the potential next-in-class triple combination medicine — will take place at the North American Cystic Fibrosis Conference (NACFC). Vertex also announced presentations describing long-term outcomes in people with cystic fibrosis (CF) ages 2 to 11 years taking TRIKAFTA®, demonstrating consistent and sustained improvements across multiple measures of disease. “TRIKAFTA has transformed the treatment of CF in the 5 years since its approval, changing the outlook for patients with CF,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “Toward our goal to bring all eligible patients to normal levels of CFTR function, we have made global regulatory submissions for our next-in-class vanza triple combination medicine for patients with CF aged 6 years and older. We are excited to bring this promising new medicine, which has the potential to deliver even greater improvements in sweat chloride, to patients with CF.” Data on the Investigational Once Daily Next-in-Class Triple Combination Therapy, vanzacaftor/tezacaftor/deutivacaftor Vertex will present data on the vanza triple in an oral presentation and two posters on Friday, September 27. This is the first time that the clinical data from the Phase 3 clinical trials of the vanza triple in patients 6 years and older with CF have been accepted for presentation at a medical meeting. These data formed the basis of global regulatory submissions. “The additional, significant reductions in sweat chloride we see in the vanza triple clinical trials are noteworthy. I believe this improvement in CFTR function may lead to important benefits for people with CF,” said Claire L. Keating, M.D., Co-Director of the Gunnar Esiason Adult Cystic Fibrosis and Lung Program at Colum...

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