European Commission formally approves triple therapy

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The European Commission has formally granted a licence to Kaftrio, the first triple-combination therapy to tackle the underlying cause of cystic fibrosis (CF), bringing hope to thousands of people living with CF across the UK and beyond.

In June the European Medicines Agency (EMA) announced it was recommending the drug receive a licence for people aged 12 and over with two copies of the F508del or one copy of F508del and one copy from a number of ‘minimal function mutations’.

Less than a week later Simon Stevens, Chief Executive of NHS England, announced that on the day the drug received its formal licence, it would be made available in on the NHS in England for everyone covered by the licence, and that clinicians would be free to prescribe modulators in the portfolio for certain people with other rare mutations covered by the US Food & Drug Administration’s licensing decisions. 

Since then, similar agreements have been announced by the health bodies in Wales, Northern Ireland and Scotland. We continue to seek more information and updates about negotiations and access in the Crown Dependencies of Jersey, Guernsey, and the Isle of Man.

These breakthroughs reflect the hard work and tireless campaigning of the CF community together with the Trust over the past four years, keeping access to life-saving drugs at the top of the political agenda.

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 UK CF Registry will continue to support access to and monitoring of these new therapies for people with cystic fibrosis. It’s thanks to a huge community effort that these medicines are now available for so many people with cystic fibrosis in the UK, and as quickly as possible.

“At such a positive moment, we are also mindful that much work still remains to find drugs appropriate for all people with cystic fibrosis and further breakthroughs that will support everyone to truly live a life unlimited.”

Alexandra Andrews, 45, from Nottingham, began taking Kaftrio earlier this year on compassionate grounds. She said: “I’m so happy that others with CF all over the UK are now going to be able to benefit from this. It’s given me a new lease of life and has meant that I can do things that I just couldn’t do before.”

Shona Young, 25, from Glasgow, who is eligible for the groundbreaking new treatment, said: “I used to worry about what would happen to me as I got older but Kaftrio is going to allow me to live life to the full for even longer. It’s such an incredible and special moment for so many people with cystic fibrosis.”   

There have been some momentous changes in the treatment of CF but there is still so much more that we need to do to ensure everyone gets access to life-changing treatment and support. Your ongoing help and fundraising are vital to ensure we can continue our work for everyone with CF, both now and in the future. If you are able, please consider making a donation today to continue to support our research.

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