Vertex Pharmaceuticals presented Phase 3 clinical data for the investigational vanzacaftor/tezacaftor/deutivacaftor triple combination therapy and new long-term data on TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor) at the North American Cystic Fibrosis Conference (NACFC). The data highlight the potential of vanzacaftor as a next-in-class treatment and the sustained benefits of TRIKAFTA® in patients with cystic fibrosis (CF).
Vanzacaftor Triple Combination Shows Promise
The Phase 3 data on the vanzacaftor triple combination demonstrated non-inferiority to TRIKAFTA® in percent predicted forced expiratory volume in one second (ppFEV1) and further improvement of CFTR function, as measured by sweat chloride. These findings formed the basis of global regulatory submissions for the vanzacaftor triple combination, which has been granted Fast Track and Orphan Drug Designations by the FDA.
Claire L. Keating, M.D., Co-Director of the Gunnar Esiason Adult Cystic Fibrosis and Lung Program at Columbia University, noted, "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."
Long-Term TRIKAFTA® Benefits Confirmed
Presentations at NACFC also described long-term outcomes in CF patients aged 2 to 11 years taking TRIKAFTA®, demonstrating consistent and sustained improvements across multiple measures of disease. These studies reinforce the sustained benefit seen in studies in older people with CF, specifically that early treatment with TRIKAFTA® is associated with sustained improvements in lung function and could also lead to improved exocrine pancreatic function over time. The data presented at NACFC highlight the safety and tolerability of TRIKAFTA®, which were generally consistent with the established safety profile.
Professor Marcus A. Mall, M.D., Professor and Chair of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center at Charité - Universitätsmedizin Berlin, stated, "I have seen first-hand the positive long-term impact that improvement of CFTR function by TRIKAFTA can have on patients’ clinical outcomes. These improvements are particularly striking for me as a physician caring for young children, where improvements in things like lung function, pancreatic function and quality of life are so meaningful."
Regulatory Progress for Vanzacaftor
Vertex also announced that Health Canada has accepted for review its New Drug Submission (NDS) for vanzacaftor/tezacaftor/deutivacaftor, a once-daily triple combination therapy for people living with cystic fibrosis (CF) ages 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Michael Siauw, General Manager at Vertex Pharmaceuticals (Canada) Incorporated, said, "Vanzacaftor raises the bar set by TRIKAFTA® by demonstrating comparable lung function improvement and additional improvements in CFTR function, as measured by sweat chloride in Phase 3 trials. Health Canada’s acceptance of the vanzacaftor submission for review brings us one step closer to providing a new treatment option, with potential for additional benefit, for eligible people living with CF."
