Trust experts welcome ‘life-transforming’ treatment for cystic fibrosis

NHS England has today announced that NHS patients in England will be some of the first people in Europe to benefit from a new treatment for cystic fibrosis (CF).

Three in five patients with the genetic condition could benefit from the ‘triple combination’ treatment produced by Vertex Pharmaceuticals, called ‘Kaftrio’, or ‘Trikafta’ in the US. The treatment tackles the underlying causes of CF by helping the lungs work effectively. 

The landmark deal, which has been negotiated ahead of a licence being granted by the European Medicines Agency, means that clinicians can start prescribing the triple treatment to English patients as soon as the licence is granted, in the coming weeks.

Commenting on the news, the directors of the Trust’s paediatric (Dr Ian Balfour-Lynn) and adult (Dr Susan Madge) CF centres, said: “Today’s announcement from NHS England is wonderful news for our CF patients, and we are thrilled that they will be among the first in Europe to benefit from this life-transforming treatment.

“This is particularly welcome news as our CF patients have been shielding at home during Covid-19. We look forward to being able to provide these medicines to eligible patients as soon as possible to help them live longer, healthier lives.”

The European licence for the triple combination therapy will cover patients 12 years and older at this stage. Younger children will continue to benefit from the previous deal struck by NHS England giving them continued access to Orkambi, Symkevi and Kalydeco, where clinically appropriate, with access to the triple therapy to follow automatically, if the European regulators approve the move.

Royal Brompton Hospital’s paediatric and adult CF centres are one of the largest in Europe and treat almost 1000 people with CF each year.