Vertex’s KAFTRIO Receives EU Approval, Brings New Hope To Younger Patients With Cystic Fibrosis

The European Commission has approved a label expansion for Vertex Pharmaceuticals' cystic fibrosis therapy KAFTRIO® (ivacaftor/tezacaftor/elexacaftor), with ivacaftor, the company announced. This newly approved label greatly expands the treatment's eligible patient population to include children two years and older with at least one non-class I mutation in the CFTR gene.

“We have been working for years to bring treatment options to all people with cystic fibrosis, including those with ultra-rare mutations. We are pleased that the European Commission has broadened the KAFTRIO indication to include all CF patients 2 years and older who have at least one non-class I mutation, ensuring that even more people living with CF can benefit from this transformative medicine,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex. 

The new expanded indication will soon be available for eligible patients in Austria, Denmark, Ireland, Norway, and Sweden because of existing reimbursement agreements and through healthcare access provisions in Germany. The Company highlighted its commitment to collaborating with health authorities across the European Union to facilitate broad and timely access to KAFTRIO for all eligible patients.