The US FDA has approved a triple combination therapy, comprising vanzacaftor, tezacaftor, and deutivacaftor, for treating cystic fibrosis in individuals aged six and older. It is indicated for cystic fibrosis patients with at least one F508del mutation or another mutation in the CFTR gene.
Clinical evidence In two clinical trials involving 971 patients aged 12 and older with CF and at least one F508del mutation, the therapy demonstrated significant improvements in forced expiratory volume outcomes and reduced sweat chloride levels through week 24 compared to a triple combination therapy, comprising elexacaftor, tezacaftor, and ivacaftor. Dosage and administration Once daily oral dosage Six to less than 12 years old with o < 40 kg – Three tablets of vanzacaftor 4 mg/tezacaftor 20 mg/deutivacaftor 50 mg o ≥ 40 kg – Two tablets of vanzacaftor 10 mg/tezacaftor 50 mg/deutivacaftor 125…
