Vertex has announced that the EMA adopted a positive opinion for the label extension of Kaftrio®, in a combination regiment with ivacaftor, 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. If approved, over 1,500 children will be eligible for a medicine that can treat the underlying cause of their disease for the first time.

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.

Cystic fibrosis is a rare, life-shortening genetic disease affecting more than 83,000 globally. Over 10,650 people in the UK have CF, the second-highest number in the world. It is a progressive, multi-organ disease affecting the lungs, liver, pancreas, GI tract, sinuses, sweat glands, and reproductive tract. The daily impact of treatment for the disease is significant, and it can take up to four or more hours involving nebulisers, physiotherapy, and up to 70 tablets a day.

Currently, there is no cure for cystic fibrosis, and half of the people in the UK with CF die before they are 31.