British patients will be among the first in Europe to be prescribed Kaftrio, which significantly improves lung function, helping people with cystic fibrosis to breathe more easily.
The treatment – known as the ‘triple combination therapy’ – was given the green light by European regulators on Friday (August 21), setting live a deal struck by NHS England to get the drug onto the frontline of patient care as soon as it was licensed.
NHS chief executive Simon Stevens announced the agreement at the Health Select Committee on June 30.
The UK Cystic Fibrosis Medical Association has described the treatment as showing evidence of being potentially ‘truly life-transforming’.
The immediate availability of Kaftrio, alongside existing treatment options previously negotiated between NHS England and Vertex Pharmaceuticals, means that most patients with cystic fibrosis – more than 7,000 people in England – can benefit from a therapy which tackles the underlying causes of the condition.
As part of the commercial deal, NHS England has also secured equivalent terms for cystic fibrosis patients in Wales, Northern Ireland and Scotland paving the way for full UK access.
Health and Care Secretary Matt Hancock said: "One of my proudest moments as health secretary was helping hundreds of children access Orkambi whose lives have been transformed by this treatment.
"In June, we secured a deal with Vertex to provide its latest wonder drug Kaftrio to patients as soon as it received a licence from the European Medicines Agency. Today, that promise becomes a reality and thousands of children with cystic fibrosis across the country can now access this treatment immediately on the NHS."
David Ramsden, chief executive of the Cystic Fibrosis Trust, said: "The licensing of Kaftrio today marks a step change in the treatment of cystic fibrosis. With agreements between Vertex and governments across the UK already in place, thousands of eligible people across the UK can now discuss with their clinical team how they can start this treatment as soon as possible.
"It’s also great news that more mutations have been added to those eligible for Kalydeco and Symkevi, giving more children and adults with CF a disease modifying treatment option."
The deal will last for four years to allow for further data to be collected to better understand the impact for patients of this treatment.
