Vertex Pharmaceuticals has received approval from the European Commission (EC) for an expanded label of Kaftrio (ivacaftor/tezacaftor/elexacaftor) combination to treat certain children with cystic fibrosis (CF).
Kaftrio in combination with ivacaftor is an oral therapy. It is designed to boost the quantity and function of the CFTR protein on the cell surface.
The commission has approved the Kaftrio in a combination regimen with ivacaftor to treat CF children aged two through five years old.
These children must have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, according to the approval.
Following regulatory approval by the EC, eligible patients in Austria, Denmark, Ireland, Norway, Latvia, and Sweden will have access to the expanded indication for Kaftrio in a combination regimen with ivacaftor due to pre-existing reimbursement agreements.
Vertex is committed to collaborating with reimbursement authorities throughout the European Union (EU) to guarantee patient access for all qualified individuals.
Under the terms of the current reimbursement agreement between Vertex and the National Health Service (NHS), the drug is available in the UK.
The children aged two years and older can receive this expanded indication for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor after the MHRA approves it on 15 November 2023.
Vertex chief medical officer and global medicines development and medical affairs EVP Carmen Bozic said: “In addition to data from clinical trials, long-term and real-world data have demonstrated the significant clinical benefit of Kaftrio in eligible people living with CF, and today’s news means that young children across Europe can now benefit from this important medicine.”
Kaftrio in combination with ivacaftor is approved in the EU to treat CF in patients aged two years and older who have at least one copy of the F508del mutation in the CFTR gene.
In January last year, the commission approved the Kaftrio in a combination regimen with ivacaftor to treat CF) in young patients aged six to 11 years.
