14 January 2022 - - The European Commission has granted approval for the label extension of Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis in patients ages six through 11 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene, US-based Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) said.

As a result of long-term reimbursement agreements in Austria, Northern Ireland and Denmark, and provisions for access in health care systems such as Germany, eligible patients in these countries will have access to the expanded indication for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor shortly following regulatory approval by the European Commission.

Vertex will continue to work with reimbursement bodies across the European Union to ensure access for all eligible patients.

This medicine has also been approved by regulatory authorities in New Zealand and in Switzerland, where it is known as Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), for people with CF ages 6 and above, and we continue to work closely with reimbursement bodies in these countries to ensure access for all eligible patients.

Cystic fibrosis is a rare, life-shortening genetic disease affecting more than 83,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract.

CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene.

Children must inherit two defective CFTR genes -- one from each parent -- to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. 

CFTR mutations lead to CF by causing the CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface.

The defective function and/or absence of CFTR protein results in poor flow of salt and water 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 lung infections and progressive lung damage that eventually leads to death for many patients.

The median age of death is in the early 30s.

In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly.

Kaftrio (ivacaftor/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.

Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface by binding to different sites on the CFTR protein.

Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane.

The combined actions of ivacaftor, tezacaftor and elexacaftor help hydrate and clear mucus from the airways.

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases.