Biotechnology company Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) reported on Friday the receipt of the marketing authorization from the European Commission (EC) for KAFTRIO in a combination regimen with ivacaftor to treat people with cystic fibrosis (CF), a progressive, disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract.
The EEC has approved the company's KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor to treat people with cystic fibrosis (CF) ages 12 years and older with 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. KAFTRIO is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface.
Upon approval of the triple combination regimen, the company 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, stated the company.
In conjunction, the EC marketing authorization was based on the company's encouraging two global Phase 3 studies, which showed statistically significant and clinically meaningful improvements in lung function (primary endpoint) in people with CF ages 12 years and older with one F508del mutation and one minimal function mutation or two F508del mutations in the CFTR gene.
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