Orphan drugs for cystic fibrosis and the hereditary rare disease hATTR are on course for European approval after winning the backing of a key regulatory advisory committee.
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.
It may be the 70th birthday of the NHS but this was no day of celebration for a group of patients with cystic fibrosis who have been told by officials there is no way forward in a pricing r
UK patients are stepping up efforts to resolve the “heartbreaking” row that is denying their families access to Vertex’s groundbreaking cystic fibrosis (CF) drug Orkambi, after Sweden becam
