NHS England and Vertex break stalemate over price of CF drugs

Four years after Orkambi was approved in the European Union, this life-saving drug will now be available to Cystic Fibrosis (CF) patients in England through the NHS. The reason for the delay was continued failing pricing negotiations between Orkambi's manufacturer, Vertex, and NHS England, leading to patient advocacy campaigns supported by Labour Party leader Jeremy Corbyn. Vertex and NHS England have announced they have come to an access agreement for all three of the former’s licensed Cystic Fibrosis (CF) drugs, Orkambi (lumacaftor/ivacaftor), Symkevi (tezacaftor/ivacaftor) and Kalydeco (ivacaftor). This means within 30 days NHS England physicians will be able to prescribe these drugs for eligible patients. Currently, this totals around 5,000 patients based on the approved indications of the CF drugs, but there is no limit to the number of patients as the access agreement is linked with all future indications of Orkambi, Symkevi and Kalydeco. Orkambi will be available to all patients over the age of two with two copies of the F508del mutation in the Cystic Fibrosis transmembrane conductance regulator (CFTR) gene, while Symkevi is indicated for patients aged 12 or older with either two copies of F508del mutation in the CFTR gene or one F508de mutation and another of 14 other mutations in the same gene.