Vertex has announced that the EMA adopted a positive opinion for extension of Kaftrio for the treatment of cystic fibrosis (CF) in patients aged 6 to 11 years old.

Those eligible will have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. 

In those with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, which can prevent the CFTR protein from reaching the cell surface and functioning properly. Kaftrio (ivavaftor/tezacaftor/elexacaftor) in combination with ivacaftor is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface.

“Today marks an important milestone for the treatment of children with CF in Europe. If approved, Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor will offer physicians a new treatment option for these young patients to help combat this life-shortening condition as early as possible,” said Carmen Bozic, MD, Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex.

The opinion of the highly influential CHMP Committee of the European Medicines Agency (EMA) will now need final approval by the European Union. The extension of Kaftrio to 6-11 year olds in Ireland will then follow without the need for a health technology assessment HTA under the innovative 2017 ‘Orkambi’ Portfolio Agreement between the HSE and Vertex that is supported by Cystic Fibrosis Ireland.