NHS 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 and enhancing their overall quality of life. ... European Commission for use in combination with ivacaftor to treat people aged 12 years and older with certain forms of cystic fibrosis. © 2020 BBC. have two copies of the F508del mutation or one copy of F508del and one copy of a 'minimal function mutation'. Leicester City have been huge supporters, along with the National Literacy Trust.". Sign up to our campaigning mailing list and we'll keep you up to date on the CF campaigns you're interested in and the campaign actions relevant to you. Cystic fibrosis patients offered 'life-transforming' drug. 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. Kaftrio ®, produced by Vertex Pharmaceuticals, is a medicine that tackles the underlying causes of the disease by helping the lungs work more effectively. The drug, Kaftrio, is produced by Vertex Pharmaceuticals and contains three key ingredients: tezacaftor, ivacaftor, and elexacaftor. So said Martin Smith, one of thousands of people with cystic fibrosis (CF) thrilled at the news "life-saver" Kaftrio is to be available on the NHS. Find out what's been happening in our campaign for life-saving drugs since it began in 2015. We are seeking more information and updates about negotiations and access in the Crown Dependencies of Jersey, Guernsey, and the Isle of Man. Kaftrio, made by US firm Vertex, has been dubbed “almost a cure” for the cruel genetic condition. Mr Smith, who lives in Northampton, has one copy of F508del and a mutation. Cystic fibrosis (CF) patients can now get a "life-transforming" treatment on the NHS in England. We are seeking more information and updates about negotiations and access in the Crown Dependencies of Jersey, Guernsey, and the Isle of Man. Cystic fibrosis causes a build-up of thick, sticky mucus in the lungs, digestive system and other organs, causing a wide range of challenging symptoms affecting the entire body. Well I'm not, but this is still such good news. The European Commission has formally licensed Kaftrio for use by people with CF who: This decision by the EMA is different to the one made by the FDA in America, who allow the drug to be prescribed to people over 12 who have at least one copy of the F508del mutation. Nine in 10 people with the genetic condition - more than 7,000 in England - … The Daily Express has been campaigning with families for … I never thought it would sell. 24th August 2020 . Find out more in our privacy and cookies policy. "My daughter, Emily, eight, thought it meant I would be getting a third lung. 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. A Co Antrim woman with cystic fibrosis (CF) has welcomed news that a life-saving drug will now be available for Northern Irish sufferers of the disease. 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. .css-8h1dth-Link{font-family:ReithSans,Helvetica,Arial,freesans,sans-serif;font-weight:700;-webkit-text-decoration:none;text-decoration:none;color:#FFFFFF;}.css-8h1dth-Link:hover,.css-8h1dth-Link:focus{-webkit-text-decoration:underline;text-decoration:underline;}Read about our approach to external linking. Could Brexit make your food more expensive? The deal with NHS England included 'tag along rights' for the other devolved nations that paved the way for similar deals across the UK. A joint statement by the Cystic Fibrosis Trust and the UK Psychosocial Professionals in CF to support those in the CF community who may be facing their own issues regarding the licencing of Kaftrio. by. "You cannot stop the mucus inside you, it is never ending, it's an onslaught.". The introduction of Kaftrio for a large proportion of PWCF represents a new era in the management of CF. (England and Wales) 1079049, Registered Charity No. Kaftrio is a 'triple-combination therapy' made up of three different compounds, tezacaftor and ivacaftor (which together make up Symkevi) combined with elexacaftor. "If only this had been around years ago.". The BBC is not responsible for the content of external sites. Trikafta belongs to a class of drugs called CFTR modulators.It is designed for people with cystic fibrosis (CF) who have at least one F508del mutation in their CFTR (cystic fibrosis transmembrane conductance regulator) gene. Health Secretary Jeane Freeman, said: “Cystic fibrosis is an inherited condition, which tragically shortens lives and affects around 900 people in … Trikafta is the US name for Kaftrio, whereas the drug has the brand name Kaftrio in Europe. The Cystic Fibrosis Trust says the drug, which it described as life-saving, can be prescribed to "people aged 12 and over with two copies of the F508del mutation, or one copy of F508del and one copy of a 'minimal function mutation'.". Are universities doing enough to look after students? About 90 percent of all people with CF have this … The Cystic Fibrosis Trust and CF teams know that this is a difficult period for those who cannot take Kaftrio, and the Trust remains committed to supporting the development of new and effective treatments for all people with CF whatever their age, genotype or … 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. The medicine, Kaftrio… The expected increase in lung function from Kaftrio is between 10% and 14% for those who are eligible and other potential health benefits, including significantly less exacerbations - the sudden worsening of CF leading to hospitalisation. It changed my life," he said. We are working with all key stakeholders to support access to everyone in the UK who could benefit. As I understand it, not everyone will be on the list, and that will be tough to take," he said. Kaftrio (Trikafta in the US) – Triple combination therapy Read about Vertex Pharmaceuticals' triple combination therapy Kaftrio (known as Trikafta in the United States), which combines ivacaftor, tezacaftor and elexacaftor, and find out how it could help to treat people with cystic fibrosis (CF). Following formal licensing by the European Commission, clinicians across the UK are now able to start prescribing Kaftrio to eligible patients. "We have been very excited today, there has been tears in the house," he said. NHS England and Vertex Pharmaceuticals have reached an agreement that will allow access to Vertex’s Kaftrio (ivacaftor /tezacaftor/elexacaftor), a triple combination therapy for cystic fibrosis (CF) to be used in a combination regimen with Kalydeco, as soon as the medication is approved by the European Commission. About Cystic Fibrosis. The people saving panto this Christmas - oh yes they are! In clinical trials for Kaftrio, people with two copies of the F508del mutation had a 10% increase in lung function compared to treatment with Symkevi, and people with a single copy of F508del had more than a 14% increase in lung function compared to treatment with the placebo. Read about Vertex Pharmaceuticals' triple combination therapy Kaftrio (known as Trikafta in the United States), which combines ivacaftor, tezacaftor and elexacaftor, and find out how it could help to treat people with cystic fibrosis (CF). Find out more about our campaign to ensure that people with CF across the UK can have access to innovative precision medicines. 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. You can read more about our research that leaves no one behind and donate to support this vital work. Real-world studies such as RECOVER are an important complementary source of knowledge on the impact of treatments on people living with this condition. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. Kaftrio is a medicine used to treat eligible patients aged 12 years and above who have cystic fibrosis. Despite regular stints in hospital, he worked in journalism and communications, before eventually having to leave employment in his 30s. What you need to know about vaccine safety. You can change your cookie settings at any time if you want. Mr Smith had half his right lung removed at the age of two. By continuing to use our site, you are agreeing to our use of cookies. 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 … Trikafta is a combination drug that includes three different drugs: elexacaftor, tezacaftor, and ivacaftor. Learn more about Kaftrio in our Life-saving drugs FAQs. .css-14iz86j-BoldText{font-weight:bold;}"It's difficult to manage an illness that will limit your life - so this news is mind blowing.". The former Coventry Telegraph journalist is 41, approaching what he described as the average life expectancy for people with CF. CF is degenerative, it takes over your whole life. ", He still faces a wait to see if he will be prescribed it. Kaftrio is already marketed in the United States as Trikafta, which was approved by the FDA in October 2019. People have 2 copies of this gene, one inherited from each parent and the disease only occurs when there is a mutation in both copies. The European Commission (EC) approved Kaftrio (ivacaftor / tezacaftor / elexacaftor) in combination with Kalydeco (ivacaftor) to treat cystic fibrosis (CF) in people 12 and older who have either two F508del mutations or one F508del mutation and one minimal function mutation in the CFTR gene. However, due to the high cost, clin- Boris Johnson says the UK must be able to follow its own rules, as he arrives in Brussels for talks. Cystic fibrosis latest: New wonder drug Kaftrio gives hope to sufferers Cystic fibrosis wonder drug Kaftrio is now being rolled out across the UK … The two names refer to the same drug. 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. When he gave up work, he started writing his best-selling Charlie Fry series of children's books. .css-1hlxxic-PromoLink:link{color:inherit;}.css-1hlxxic-PromoLink:visited{color:#696969;}.css-1hlxxic-PromoLink:link,.css-1hlxxic-PromoLink:visited{-webkit-text-decoration:none;text-decoration:none;}.css-1hlxxic-PromoLink:link:hover,.css-1hlxxic-PromoLink:visited:hover,.css-1hlxxic-PromoLink:link:focus,.css-1hlxxic-PromoLink:visited:focus{color:#B80000;-webkit-text-decoration:underline;text-decoration:underline;}.css-1hlxxic-PromoLink:link::after,.css-1hlxxic-PromoLink:visited::after{content:'';position:absolute;top:0;right:0;bottom:0;left:0;z-index:2;}Cystic fibrosis patients offered 'life-transforming' drug, PM: EU demands in Brexit talks 'unacceptable'. "I'm the last one (alive) out of my friends that I made at the hospital," he said. Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. Kaftrio is indicated in a combination regimen with ivacaftor 150 mg tablets for the treatment of cystic fibrosis (CF) in 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 (see section 5.1). We also support the UK CF Registry, which will continue its valuable work in monitoring the effectiveness of this drug, among many others. Clinical trials have shown that Kaftrio is life transforming for 90% of patients with the illness. It is difficult knowing that not everyone will stand to benefit from Kaftrio or the other currently licensed modulators. Kaftrio ®, produced by Vertex Pharmaceuticals, is a medicine that tackles the underlying causes of the disease by helping the lungs work more effectively. Selina McKee. VideoiPhone helps me navigate the street without sight. "I write them for Emily so she can say 'my dad did that'. "I'm hopeful but apprehensive. New cystic fibrosis drug Kaftrio on NHS is 'mind-blowing' Published 1 July. Download the factsheet. Kaftrio is a medicine used to treat patients aged 12 years and above who have cystic fibrosis, an inherited disease that has severe effects on the lungs, the digestive system and other organs. From other local news sites. Kaftrio: Turning around the lives of patients with cystic fibrosis 26 August 2020 Last week (21 August), NHS England was given the green light by the European Medicines Agency to begin prescribing the life-changing cystic fibrosis drug, Kaftrio, to patients. Kaftrio is a 'triple combination therapy' which helps improve lung function It will help those with cystic fibrosis, a hereditary disease, to breathe more easily The Trust is fully committed to finding effective treatment for everyone with cystic fibrosis. "It's kind of ironic that my books took off as my health declined. The Welsh Government has secured a deal for a so-called wonder drug which could be used to help people living with cystic fibrosis. Journal of Cystic Fibrosis 19 (2020) 688–695 Contents lists available at ScienceDirect Journal of Cystic Fibrosis ... (probably under the name of Kaftrio TM but for simplicity we will further refer to this triple combination as Trikafta TM ) is forecast for 2020. Cystic fibrosis patients offered 'life-transforming' drug, My grandad’s ‘triggering’ 1960s race documentary. The details of which precise genotypes the latter group covers is to be confirmed. The drug, Kaftrio, is produced by Vertex Pharmaceuticals and contains three key ingredients: tezacaftor, ivacaftor and elexacaftor. .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. ivacaftor, tezacaftor and elexacaftor, and find out how it could help to treat people with cystic fibrosis (CF). Now CF teams can prescribe Kaftrio, it will take time for teams to organise and roll-out the new drug. 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. ... Kaftrio… Trikafta’s early approval and launch was a significant milestone for Vertex. NHS England announced on 30 June 2020 that a deal had been agreed for Kaftrio. Published 30 June. Although Orkambi and Symkevi can stabilise cystic fibrosis symptoms, the newer drug is four times as effective at improving lung function. See answers to FAQs about cystic fibrosis, Get the latest news about cystic fibrosis, Paediatric specialist CF centres in the UK, Understanding and treating lung infections, Understanding and treating symptoms of CF, Clinical Training Fellowship Programme 2019/2020, Clinical Excellence and Innovation Awards, read more about our research that leaves no one behind. 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