NHS Cystic Fibrosis Triple Therapy Deal Ahead of EMA Licence

Nicky Broyd

June 30, 2020

Editor's note, 21 August 2020: NHS England said Kaftrio received European regulatory approval today allowing its use in the UK.   NHS ScotlandNHS Wales, and Northern Ireland's Department of Health have also announced deals to make Kaftrio available.

NHS England has signed a deal to make a 'transformational' triple drug combination treatment available to UK cystic fibrosis patients as soon as its licence is granted by the European Medicines Agency (EMA). 

Vertex Pharmaceuticals' new drug combines ivacaftor, tezacaftor, and elexacaftor and is expected to benefit around 7000 people with the condition. 

The drug, called Kaftrio, (Trikafta in the US) was approved by the US FDA last October.

'Landmark Deal'

Although NHS England negotiated the deal, it is supported by the National Institute for Health and Care Excellence (NICE), and the same terms will be available to the NHS in Scotland, Wales, and Northern Ireland. The Welsh Government said its discussions with the manufacturer "are progressing constructively". 

In a news release, NHS England Chief Executive Sir Simon Stevens said: "This is a potentially transformational treatment for cystic fibrosis, and I am proud that, thanks to this landmark deal, NHS patients will be among the first in Europe to benefit."

NICE will work with Vertex to continue NHS data collection and prices may be adjusted after a full NICE appraisal.

The EMA gave the drug a positive opinion on 25th June. The agency said the drug can provide significant improvements in lung function as measured by ppFEV1 (percent predicted FEV1) (14.3 percentage point reduction in heterozygous MF and 10 percentage point reduction in homozygous patients) and to reduce sweat chloride (a reduction of 41.8 mmol/L in MF and 45.1 mmol/L in homozygous patients).

The drug is for cystic fibrosis patients aged 12 years and older who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or heterozygous for F508del in the CFTR gene with a minimal function (MF) mutation.

'Life Saving'

Ludovic Fenaux, senior vice president, Vertex International, commented: "Establishing this agreement, in advance of the medicine being licensed, illustrates the collaboration between Vertex, NHS England and NICE. It shows just what is possible when the biotech industry and the NHS partner together for the benefit of patients."

NICE Chief Executive, Professor Gillian Leng, said in a statement: "NICE welcomes this agreement which will ensure people with cystic fibrosis can access this important new treatment while further data on its benefits are collected. We look forward to working with the company and the NHS on continued data collection for all three products involved in advance of our formal appraisal process."

The Cystic Fibrosis Trust's chief executive David Ramsden described the deal as "fantastic news", adding: "This will truly save lives."


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