A new EMA approval has expanded the use of Vertex Pharma’s cystic fibrosis therapy Symkevi to children as young as six if they have specific gene mutations.
Vertex remains at loggerheads with NHS England over funding for Orkambi, but said it would consider the body’s most recent offer of a £500 million, five-year funding deal.
Disc Medicine has charted a route to market for bitopertin, its drug candidate for rare disease erythropoietic protoporphyria (EPP), after meeting with the FDA.