Cystic fibrosis drug Kaftrio to be available on NHS after European licence granted

  • 8/21/2020
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The European commission has licensed a new, potentially lifesaving drug to treat cystic fibrosis, meaning it will be available on the NHS to many who have the condition. The drug, Kaftrio, is produced by Vertex Pharmaceuticals and contains three key ingredients: tezacaftor, ivacaftor and elexacaftor. Clinical trials have shown Kaftrio can increase lung function by between 10% to 14% in people with cystic fibrosis, depending on their genetic makeup, and can improve quality of life. It was announced in July that NHS England had signed a deal with Vertex to secure access to the drug, and health bodies in the other UK nations reached similar agreements soon after. The latest development means Kaftrio is now available on the NHS. Only those aged 12 and over and who have particular genetic variants will be eligible for the drug, including those with two copies of the variant F508del or one copy together with another mutation from a list that is still being refined. The NHS has said that about 7,000 people in England are expected to be eligible for the drug. Recent estimates suggest there are about 7,700 people in England with the condition. Another 300 people in England with cystic fibrosis but with rare mutations will benefit from other Vertex pharmaceutical drugs secured in the deal. The agreement has been supported by the National Institute for Health and Care Excellence (Nice), which will use data collected over its four-year period to explore the impact of the drug on patients and assess its cost-effectiveness. Shona Young, 25, from Glasgow and among those who will be able to receive Kaftrio, 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,” she said. David Ramsden, the chief executive of the Cystic Fibrosis Trust, said the latest development marked a step change in the treatment of the condition. “With agreements between Vertex and governments across the UK already in place, thousands of eligible people can now discuss with their clinical team how they can start this treatment as soon as possible,” he said. Prof Stephen Powis, the national medical director for NHS England, said the deal has put patients “in the front of the queue” for the treatment. “The NHS has developed world-leading commercial capabilities, enabling us to strike such landmark deals with the pharmaceutical industry and support people with cystic fibrosis to lead longer, healthier, better quality lives,” he said.

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