Vertex Presents Promising Phase 3 Data on CF Therapy at NACFC

Vertex Pharmaceuticals (Nasdaq: VRTX) has announced the first medical presentations of Phase 3 data on its investigational once-daily triple combination therapy, vanzacaftor/tezacaftor/deutivacaftor, at the North American Cystic Fibrosis Conference (NACFC). This new treatment, referred to as the “vanza triple,” aims to become a next-in-class option for cystic fibrosis (CF) patients, aged six years and older. Vertex has made global regulatory submissions for the vanza triple, with a Prescription Drug User Fee Act (PDUFA) date set for January 2, 2025, by the U.S. FDA.

Dr. Carmen Bozic, Executive Vice President of Global Medicines Development and Chief Medical Officer at Vertex, emphasized that the vanza triple has the potential to further improve CFTR function and reduce sweat chloride levels in CF patients, building on the progress made by TRIKAFTA®.

Key Phase 3 data will be presented in both oral and poster sessions at NACFC, marking the first time these results have been shared at a medical conference. Clinicians, including Dr. Claire L. Keating of Columbia University, are optimistic that vanza triple’s once-daily dosing and improved CFTR functionality will offer meaningful advancements for CF treatment.

Alongside the vanza triple data, Vertex will also present new long-term findings on TRIKAFTA®, its transformative CF treatment. Studies of children aged 2 to 11 years old demonstrate sustained improvements in lung and pancreatic function, reinforcing the benefits of early intervention with TRIKAFTA.

Additionally, Vertex will share real-world evidence from TRIKAFTA and KALYDECO® therapies, showcasing their efficacy and safety across multiple age groups and CF genotypes. With its medicines already treating over 68,000 people with CF globally, Vertex continues to lead the charge in developing life-changing therapies for CF patients.