Cystic fibrosis: What next for the “wonder drug” Kaftrio?

What is Kaftrio?The management of patients with cystic fibrosis has been transformed over the past few years by the advent of treatments designed to correct the malfunctioning protein made by the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Kaftrio is the latest of these CFTR modulator treatments and consists of a triple treatment combination of ivacaftor, tezacaftor, and elexacaftor. It’s a long term treatment for patients with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene—around 90% of people with CF.In 2019, after a lengthy battle over cost, an agreement was reached between NHS England and the drug company Vertex to make two earlier CFTR modulator treatments—Orkambi (lumacaftor-ivacaftor) and Symkevi (ivacaftor-tezacaftor)—available on the NHS.12 As part of the interim access agreement NHS England, the National Institute for Health and Care Excellence (NICE), the UK Cystic Fibrosis Trust, and Vertex would collect and share real world…
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