FDA approves next-generation triple therapy for cystic fibrosis

NEWS IN BRIEF 13 January 2025

In December, the FDA approved Vertex’s vanzacaftor, tezacaftor and deutivacaftor (Alyftrek) for the treatment of cystic fibrosis in patients with at least one F508del mutation or another treatment-responsive mutation in the CFTR gene. The fixed-dose triple-combination therapy, dubbed ‘vanza triple’, is set to build on the success of Vertex’s top-selling combination of elexacaftor, tezacaftor and ivacaftor (Trikafta), which was approved by the FDA in 2019 and is on track to earn more than US$10 billion in annual sales. Whereas patients have to take the older triple combination twice a day, they only have to take the new one once a day.

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