EU nod for Vertex CF therapy Kaftrio

Vertex Pharmaceuticals' Kaftrio (ivacaftor/tezacaftor/elexacaftor) has been approved by the European Commission for use in combination with ivacaftor to treat people aged 12 years and older with certain forms of cystic fibrosis.

Specifically, the decision allows doctors to prescribe the drug when people with the disease are carrying one F508del mutation and one minimal function mutation (F/MF), or two F508del mutations (F/F) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

It means that, for the first time, up to 10,000 people in Europe ages 12 years and older with CF and these mutations will be eligible for a CFTR modulator that treats the underlying cause of the disease.

Approval of the triple combination regimen also expands the number of treatment options available to people ages 12 years and older with CF who have two copies of the F508del mutation, the most common CF-causing mutation worldwide, the firm noted.

Clearance was based on data from two global Phase III studies, which showed statistically significant and clinically meaningful improvements in lung function (primary endpoint) and all key secondary endpoints.

“The triple combination regimen has been shown to have a major impact on several outcome measures in people with CF,” said Professor Harry Heijerman, Professor and head of the Department of Pulmonology at University Medical Center Utrecht, Netherlands. “The clinical data showed significant improvements in lung function and other important measures, such as sweat chloride levels and quality of life as measured by the CFQ-R respiratory domain score, in patients treated with the triple combination therapy.”

David Ramsden, chief executive of the Cystic Fibrosis Trust, said the approval “marks a step change in the treatment of cystic fibrosis”, adding: “It’s also great news that more mutations have been added to those eligible for Kalydeco and Symkevi, giving more children and adults with CF a disease modifying treatment option.”

Long-term reimbursement agreements for the triple combination have already been secured in the UK, meaning that eligible patients will now have access to the treatment.

The terms of the deal landed by NHS England also mean that as many as 300 patients with some rarer genetic mutations, which fall outside of the scope of the European licensing body’s considerations, will now be able to benefit from Kalydeco and Symkevi, also produced by Vertex Pharmaceuticals, offering a lifeline to those with limited treatment options.