BRIEF—Expanded Orkambi approval gives access to select group in UK

US drugmaker Vertex Pharmaceuticals has announced that the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has approved an expanded use of Orkambi (lumacaftor/ivacaftor).

The approval is for the treatment of children in England, Scotland and Wales with cystic fibrosis (CF) aged one to two years who have two F508del mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Andrew Wilfin, senior medical director, Vertex UK and Ireland, said: “This approval is great news for the approximately 80 children living with CF who are now eligible for a medicine to treat the underlying cause of their disease.

“It is another important step on our journey to reach people of all ages living with CF who may benefit from our medicines.”

Access for these patients is included in the long-term reimbursement agreements in place between Vertex and the National Health Service in England, Scotland and Wales.