The Daily Express has been campaigning with families for … We have produced a factsheet on the emotional and social impact impacts of Kaftrio, for those who are eligible for the treatment, as well as those who will not be able to benefit from it. Kaftrio is a medicine used to treat eligible patients aged 12 years and above who have cystic fibrosis. The drug generated sales worth $1.8 billion in the first half of 2020. Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is degenerative, it takes over your whole life. Please click here for more information on the RECOVER study. The drug, Kaftrio, is produced by Vertex Pharmaceuticals and contains three key ingredients: tezacaftor, ivacaftor and elexacaftor. The medicine will be available for eligible patients with Cystic Fibrosis (CF) from the point at which it is granted its European marketing authorisation later this summer. Clinical trials have shown that Kaftrio can increase lung function by 10% to 14% in people with cystic fibrosis, depending on their genetic makeup, and can improve quality of life. From today thousands of NHS cystic fibrosis patients in England can benefit from a ‘transformative’ treatment for cystic fibrosis. The deal with NHS England included 'tag along rights' for the other devolved nations that paved the way for similar deals across the UK. .css-po6dm6-ItalicText{font-style:italic;}Follow BBC West Midlands on Facebook, on Twitter, and sign up for local news updates direct to your phone. ... European Commission for use in combination with ivacaftor to treat people aged 12 years and older with certain forms of cystic fibrosis. Now CF teams can prescribe Kaftrio, it will take time for teams to organise and roll-out the new drug. NHS England announced on 30 June 2020 that a deal had been agreed for Kaftrio. Selina McKee. Kaftrio is the first drug that works in patients with the F508del and MF mutations, who account for around 90% of the overall CF population, and … I never thought it would sell. By continuing to use our site, you are agreeing to our use of cookies. (Scotland) SC040196. Real-world studies such as RECOVER are an important complementary source of knowledge on the impact of treatments on people living with this condition. VideoMy grandad’s ‘triggering’ 1960s race documentary, Beavers, burpees and bread: 2020's most-searched, 'How my foot became anti-vaccine propaganda', iPhone helps me navigate the street without sight. "My daughter, Emily, eight, thought it meant I would be getting a third lung. This was followed by similar deals in Wales on 22 July, in Northern Ireland on 30 July and in Scotland on 4 August,  with Vertex to facilitate access from the first day the European licence is granted. And while Orkambi and … We are working with all key stakeholders to support access to everyone in the UK who could benefit. Kaftrio is approved in Europe for the treatment of cystic fibrosis (CF) in patients ages 12 years and older with one F508del mutation and one minimal function mutation or two F508del mutations in the CFTR gene. So said Martin Smith, one of thousands of people with cystic fibrosis (CF) thrilled at the news "life-saver" .css-yidnqd-InlineLink:link{color:#3F3F42;}.css-yidnqd-InlineLink:visited{color:#696969;}.css-yidnqd-InlineLink:link,.css-yidnqd-InlineLink:visited{font-weight:bolder;border-bottom:1px solid #BABABA;-webkit-text-decoration:none;text-decoration:none;}.css-yidnqd-InlineLink:link:hover,.css-yidnqd-InlineLink:visited:hover,.css-yidnqd-InlineLink:link:focus,.css-yidnqd-InlineLink:visited:focus{border-bottom-color:currentcolor;border-bottom-width:2px;color:#B80000;}@supports (text-underline-offset:0.25em){.css-yidnqd-InlineLink:link,.css-yidnqd-InlineLink:visited{border-bottom:none;-webkit-text-decoration:underline #BABABA;text-decoration:underline #BABABA;-webkit-text-decoration-thickness:1px;text-decoration-thickness:1px;-webkit-text-decoration-skip-ink:none;text-decoration-skip-ink:none;text-underline-offset:0.25em;}.css-yidnqd-InlineLink:link:hover,.css-yidnqd-InlineLink:visited:hover,.css-yidnqd-InlineLink:link:focus,.css-yidnqd-InlineLink:visited:focus{-webkit-text-decoration-color:currentcolor;text-decoration-color:currentcolor;-webkit-text-decoration-thickness:2px;text-decoration-thickness:2px;color:#B80000;}}Kaftrio is to be available on the NHS.