Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval of the label extension for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in all patients ages 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 12 years and older who are heterozygous for the F508del-CFTR mutation and a residual function (F/RF) or gating mutation (F/G) are eligible for the triple combination therapy for the first time, and the majority of people ages 12 years and older with CF in Europe are now eligible for KAFTRIO.

“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, M.D., Chief Executive 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.”

In both the United States and Australia, where the triple combination therapy is known as TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), it is already licensed for the treatment of people with CF ages 12 years and older who have at least one copy of the F508del mutation in the CFTR gene, regardless of the other mutation type.

“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’ (F/G) or ‘residual function’ (F/RF) 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,” said Dr Peter Barry, Honorary Senior Lecturer at The University of Manchester.

As a result of long-term reimbursement agreements in the Republic of Ireland, Northern Ireland, Denmark and Luxembourg, and provisions for access in health care systems such as Germany, eligible patients in these countries will have access to the expanded indication for the triple combination regimen shortly following regulatory approval by the European Commission.