Vertex

Mother helps child to use a nebuliser

EU extends paediatric use of combination in cystic fibrosis

27 November 2023

A label expansion for Kaftrio (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) has been approved in the European Union (EU). The European Commission has extended use of the therapy to include children with CF aged two through five years old who have at least one F508del mutation in […]

CRISPR gene editing

Great Britain first to approve CRISPR-based gene therapy

16 November 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Casgevy (exagamglogene autotemcel, or exa-cel) is the first medicine to be licensed that uses gene editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020. The treatment from […]