Vertex’s ALYFTREK® Approved In EU, Demonstrates Stronger CFTR Modulation For Expanded Cystic Fibrosis Treatment

Vertex Pharmaceuticals has received approval from the European Commission for ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor), a next-generation triple combination therapy for treating cystic fibrosis (CF) in patients aged 6 and older with at least one non-class I CFTR gene mutation. This milestone expands Vertex’s reach in CF care across the EU.

“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. 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,” 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. 

In pivotal head-to-head clinical trials, ALYFTREK® was shown to be non-inferior to Vertex’s existing therapy KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in improving lung function (ppFEV1), while outperforming it in reducing sweat chloride levels, a key biomarker of CFTR function.

“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. With this approval, we are one step closer to our ultimate goal of restoring normal levels of CFTR function in people living with CF,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. 

Thanks to established reimbursement agreements in countries such as Ireland and Denmark, as well as early-access frameworks in Germany, eligible CF patients in these regions will have timely access to the new therapy. Vertex is actively engaging with additional EU member states to secure broader reimbursement and access across Europe.