Press release
Vertex to Present Long-Term Data Demonstrating Significant Benefits of Treatment With CFTR Modulators at North American Cystic Fibrosis Conference (NACFC)
- 96-week interim results of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) study show no loss of pulmonary function in people with at least one
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n- 96-week interim results of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) study show no loss of pulmonary function in people with at least one F508del allele, a first for any CFTR modulator –\n\n- Real-world data from people treated with KALYDECO® (ivacaftor) over approximately 6 years show lower rates of mortality, lung transplant and pulmonary exacerbations than comparator cohort -\n\n- Additional presentations highlight safety and efficacy profile of TRIKAFTA® -\n\n BOSTON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that five scientific abstracts about the company’s portfolio of cystic fibrosis (CF) medicines will be presented at the 2021 North American Cystic Fibrosis Conference (NACFC) taking place virtually November 2-5, 2021.\n\nKey data being presented include 96-week interim results from an ongoing TRIKAFTA® open-label extension study in people with CF ages 12 years and older with F508del/Minimal Function (F/MF) or F508del/F508del (F/F) genotypes, showing that the favorable safety profile and clinically meaningful improvements in lung function, respiratory symptoms and CFTR function as measured by sweat chloride observed in the Phase 3 pivotal studies were maintained through an additional 96 weeks of treatment (Poster #681). Additionally, a post hoc analysis of the annualized mean rate of change in percent predicted forced expiratory volume in 1 second (ppFEV1) showed there was no loss of pulmonary function over 96 weeks in this CF population, which is a first for any CFTR modulator to date.\n\nAlso presented at this year’s conference are data on results from a retrospective study of patients with gating mutations ages 6 years or older treated with KALYDECO® showing that people treated with KALYDECO® over approximately six years of follow up had significantly lower rates of mortality, lung transplant and pulmonary exacerbations (PEx) compared to a cohort of patients that were not eligible for and not receiving KALYDECO treatment (Poster #178).\n\n“The data we’re presenting this year clearly demonstrate that our portfolio of CFTR modulators has truly transformed the CF treatment landscape,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. “The long-term follow up data from TR...