Vertex Pharmaceuticals’ cystic fibrosis (CF) therapy Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) has been approved by the European Commission (EC).
The triple combination treatment has been specifically authorised for use in CF patients aged six years and older who have at least one non-class I mutation in the CF transmembrane conductance regulator (CFTR) gene.
Affecting more than 109,000 people worldwide, CF is a rare inherited disease caused by a faulty CFTR gene, which helps regulate the flow of water and chloride into and out of the cells in a number of organs.
In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic infections and progressive damage that eventually leads to death for many patients.
Taken orally once daily, Vertex’s Alyftrek is a CFTR modulator that works by correcting the malfunctioning protein made by the CFTR gene.
The EC’s decision on the drug was supported by results from two head-to-head clinical trials, which showed that Alyftrek was as effective at improving lung function as Vertex’s currently approved triple combination therapy Kaftrio (ivacaftor/tezacaftor/elexacaftor), and more effective at reducing sweat chloride levels.
Vertex’s chief executive officer and president, Reshma Kewalramani, said: “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.”
Reimbursement agreements in Ireland and Denmark, and provisions for access in healthcare systems such as Germany, will allow eligible patients in these countries to benefit from Alyftrek shortly, while Vertex said it will continue to work with reimbursement bodies across the EU member states to ensure access for all eligible patients “as quickly as possible”.
Welcoming the approval, Marcus Mall, professor and chair of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center at Charité Universitätsmedizin Berlin, said: “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.
“[Alyftrek] 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.”
