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EC approves Kaftrio as combination treatment for cystic fibrosis

Published on 29/04/21 at 09:37am

Vertex Pharmaceuticals has been granted European Commission approval for the label extension of Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor, for the treatment of cystic fibrosis (CF) in all patients aged 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

With this extension of the indication, CF patients who are heterozygous for the F508del-CFTR mutation and a residual function or gating mutation, are eligible for the triple combination therapy for the first time.

Reshma Kewalramani, MD, Chief Executive Officer and President at Vertex, said: “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.

“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.”

The triple combination therapy is already available to patients in the US and Australia, and is known as Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor).

Dr Peter Barry, Honorary Senior Lecturer at The University of Manchester, said: “In clinical trials ivacaftor/tezacaftor/elexacaftor plus ivacaftor showed positive significant results in people with cystic fibrosis ages 12 years and older who have at least one copy of the most common gene mutation (F508del) with the latest results showing clinical benefits in individuals who have an additional ‘gating’ or ‘residual function’ gene mutation.

“It is welcome news for the CF community that the European regulatory authority has extended the licensed indications for this therapy based on the latest trial results which means that additional patients will gain access to this medication.”

CF is a rare, life-shortening, genetic disease affecting more than 80,000 people globally. It is a progressive, multi-system disease that affects the lungs, liver, gastrointestinal tract, sinuses, sweat glands, pancreas, and reproductive tract.

CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene, while there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation.

Ivacaftor/tezacaftor/elexacaftor has been designed to increase the quantity and function of the F508del-CFTR protein at the cell surface.

Kat Jenkins

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