Vertex's innovative triple therapy Kaftrio has been approved by the European Union

recently announced that Vertex Pharmaceuticals has announced that the European Commission (EC) has approved kaftrio(ivacaftor/tezacaftor/elexacaftor) and Kalydeco (ivacaftor) 150mg joint drug treatment for age ≥ 12-year-old CF patients, specifically: patients with an F508del mutation and a minimal functional mutation (F/MF) in the cystic fibrosis transfilm conductivity regulator (CFTR) gene, or 2 patients with an F508del mutation (F/F).this approval, for the first time ever, up to 10,000 patients in Europe ≥12 years of age, with an F508del mutation and a minimal functional mutation, are eligible for a treatment that targets the underlying causes of the disease in addition, CF patients aged ≥12 years with two F508 mutations will also be eligible for a new triple treatment, which is currently eligible for another CF drug approved by Vertex.the U.S., the triple therapy was approved by the FDA in October 2019 under the brand name Trikafta (elexacaftor/tezacaftor/ivacaftor-ivacaftor) for: CFT There is at least one patient in the R gene with an F508del mutation, aged ≥12 years, specifically in patients with one F508del mutation and one minimal functional mutation, or with two F508del mutations. Previously, the FDA had granted Trikafta priority qualification, fast-track eligibility, and breakthrough drug eligibility (BTD).The F508del mutation is the most common mutation that causes CF. In the U.S., Trikafta's approval is the first time that about 6,000 patients ≥12 years old, with an F508del mutation and a minimal functional mutation (F/MF) have a treatment for the underlying cause of the disease. In addition, about 12,000 CF patients with one or two F508del mutations who are eligible for Vertex's three other FDA-approved CF medications are also eligible for Trikafta treatment.the European Union approved Kaftrio based on the results of two Global Phase III studies: a 24-week placebo-controlled study in patients aged ≥12 years, with an F508del mutation and a minimal functional mutation; The head study, conducted in CF patients aged ≥ 12 and carrying two F508del mutations, compared triple therapy (ivacaftor/tezacaftor/elexacaftor) with tezacaftor/ivacaftor. Both studies showed statistically and clinically significant improvements in the main endpoints (lung function, ppFEV1) and all key secondary endpoints. In both studies, the ivacaftor/tezacaftor/elexacaftor and ivacaftor combined drug treatment programs were well-to-do.fibrosis (CF) is a rare, life-shortening genetic disease that affects about 75,000 people worldwide. CF 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 defect or deficiency of the CFTR protein caused by certain mutations in the CFTR gene. Children must inherit two defective CFTR genes (one for each parent) to develop CF.there are many different types of CFTR mutations that can cause disease, the vast majority of CF patients have at least one F508del mutation. These mutations can be determined by genetic testing or genotyping. CFTR proteins usually regulate the transport of ions in cell membranes, and genetic mutations can lead to the destruction or loss of protein product function. When cell membrane ion transport is interrupted, the viscosity of the mucus coating of some organs thickens. A major feature of the disease is the accumulation of thick mucus in the respiratory tract, which leads to difficulty breathing, chronic recurrent lung infections and recurrent lung damage, which eventually leads to death. The median age of death for CF patients is in their mid-30s., Vertex currently lists several CF drugs: Kalydeco( ivacaftor), Orkabi (lumacaftor/ivacaftor) and Symkevi/Symdeko (tezacaftor/iafvactor) can treat about 40,000 patients worldwide, or about 50 percent of all CF patients. The new triple therapy Trikafta could expand the treatment to 90% of CF patients worldwide. (Bio Valley)original title: Cystic Fibrosis (CF) Breakthrough Drug! Vertex's innovative triple therapy Kaftrio has been approved by the European Union to treat up to 90% of patients!