Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Commission has granted approval for ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor)for the treatment of people with cystic fibrosis (CF) ages 6 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
“Thousands of people with CF across the EU may now benefit from this new, once-daily medicine, which has demonstrated further improvement in CFTR protein function versus KAFTRIO,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “With this approval, we are one step closer to our ultimate goal of restoring normal levels of CFTR function in people living with CF.”
In two head-to-head pivotal clinical trials, deutivacaftor/tezacaftor/vanzacaftor was non-inferior to KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor on ppFEV1 and superior at reducing sweat chloride, demonstrating greater improvement in CFTR function.
“CF care has been transformed by the advent of highly effective CFTR modulators, and I am very pleased that we now have a new treatment option to even better address this multi-systemic disease,” said 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. “Deutivacaftor/tezacaftor/vanzacaftor has shown it can deliver greater reductions in sweat chloride compared to standard of care. By bringing more people closer to normal level of CFTR function, this new medicine has the potential to further improve outcomes for patients.”
As a result of reimbursement agreements in Ireland and Denmark and provisions for access in health care systems such as Germany, eligible patients in these countries will have access to deutivacaftor/tezacaftor/vanzacaftor shortly following regulatory approval by the European Commission. Vertex will continue to work with reimbursement bodies across the European Union member states to ensure access for all eligible patients as quickly as possible.
