Vanzacaftor/tezacaftor/deutivacaftor

The combination is indicated for the treatment of cystic fibrosis in people aged six years of age and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. == Adverse effects ==

The US Food and Drug Administration (FDA) prescription label for the combination contains a boxed warning about drug-induced liver injury and liver failure. == History ==

The US Food and Drug Administration (FDA) granted the application for vanzacaftor, tezacaftor, and deutivacaftor combination therapy orphan drug designation. == Society and culture ==

Legal status The combination was approved for medical use in the United States in December 2024. In April 2025, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Alyftrek, intended for the treatment of cystic fibrosis in people aged six years of age and older who have at least one non‑class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The combination was authorized for medical use in the European Union in June 2025. Names The combination is sold under the brand name Alyftrek. == References ==