News & Comment

First disease-modifying drug approved in the United States for the treatment of cystic fibrosis in patients with a specific mutation in the gene encoding the cystic fibrosis transmembrane conductance regulator.

Fusion protein that inhibits vascular endothelial growth factor signalling approved in the United States for the treatment of neovascular age-related macular degeneration.

First-in-class drug that inhibits protein misfolding approved in Europe for the treatment of familial amyloid polyneuropathy.

Janus kinase inhibitor approved by the US Food and Drug Administration for the treatment of myelofibrosis.

Antibody–drug conjugate specific for CD30 approved by the US Food and Drug Administration for the treatment of two types of lymphoma.