Triple combo cystic fibrosis therapy Kaftrio scores EU label expansion

The European Commission (EC) has approved a label extension for Vertex’s triple combination cystic fibrosis (CF) treatment Kaftrio, extending its use for the ‘majority’ of patients aged 12 years and older in Europe.

The label expansion means that Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor is now available for the treatment of all CF patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

This latest indication approval was supported by results of three Phase III studies in people aged 12 years and older with CF, which evaluated the drug in CF patients with a range of mutations.

In a statement, Vertex said eligible patients in the Republic of Ireland, Northern Ireland, Denmark and Luxembourg will have access to the expanded indication for Kaftrio thanks to long-term reimbursement agreements.

In Germany, patients will also shortly be able to access the treatment as a result of provisions for access in health care systems.

In Great Britain, Vertex has utilised the EC decision reliance procedure – when approved by the Medicines and Healthcare products Regulatory Agency (MHRA), the long-term reimbursement agreements in place in England, Scotland and Wales will ensure patients can access the expanded indication.

“This indication extension is important as CF patients in Europe with gating and residual function mutations will have access to Kaftrio for the first time,” said Reshma Kewalramani, chief rxecutive officer and president at Vertex.

“We look forward to working with health authorities and governments to ensure that all eligible patients who can benefit from this medicine have access as soon as possible,” he added.