Hundreds of cystic fibrosis patients become eligible for ‘life-changing' therapy
CF is caused by a faulty gene that affects the production of a protein called CFTR.
Modulator drugs work by helping to make the CFTR protein work effectively.
NHS officials said the treatments can 'transform' life expectancy and quality of life for patients.
There are a number of modulator therapies available but some patients with the rarest forms of the disease have been unable to access them until now.
Nice approved Alyftrek, which is known as a triple therapy, for patients aged six and over for the most common form of CF as an alternative to another treatment called Kaftrio.
And NHS England announced that the treatment would also be made available for children and adults with rare forms of cystic fibrosis, who have not previously been eligible for modulator therapy.
NHS England said the move allows patients with the rarest form of the disease to access the latest treatments, including Alyftrek and Kaftrio, for the first time.
It said that 95% of people with cystic fibrosis in England will now be eligible for modulator therapy.
The new drug, also known as vanza triple, has been found to be as effective as current drug Kaftrio in clinical trials in improving and maintaining lung function in people with CF.
The Medicines and Healthcare products Regulatory Agency (MHRA) approved the triple combination medicine – which is also known as deutivacaftor/tezacaftor/vanzacaftor – for use in the UK, for certain patients with CF over the age of six, in March.
Cystic fibrosis is a genetic condition that causes thick mucus to build up in the lungs and digestive system which leads to breathing difficulties and serious infections.
John Stewart, NHS England's director for specialised commissioning, said: 'This is a major leap forward for hundreds of patients living with the rarest forms of cystic fibrosis, offering fresh hope of a better quality of life.
'Access to a once-daily treatment at home can make an enormous difference to patients and their families – reducing the burden of hospital appointments and allowing children and young people to live more freely and independently.
'For those living with the rarest forms of the condition, this represents the very first time they will be able to access this new standard of care that has been so transformative for many since 2019.
'The rollout of this life-changing therapy demonstrates how the NHS continues to embrace innovation to deliver significant improvements in care for patients across the country at a fair price for the taxpayer.'
Helen Knight, director of medicines evaluation at Nice, said: 'CFTR modulators are already revolutionising the way cystic fibrosis is treated so we're pleased to be able to recommend Alyftrek, the latest of this type of treatment that has been shown to be effective, with significant benefits for people with the condition.'
David Ramsden, chief executive of the Cystic Fibrosis Trust, said: 'Today's announcements are another positive step in the journey to better treatments for everyone with cystic fibrosis – a lifelong, life-limiting condition without a cure.
'It's thanks to the incredible support and many years of campaigning of the CF community, and the work of all our partners, that modulator drugs are now a treatment option for thousands of people.'
Ludovic Fenaux, senior vice president at Vertex International, said the move 'represents a significant milestone in our journey to serially innovate and further improve the lives of people living with this disease'.
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
The Independent
18 minutes ago
- The Independent
New research exposes the risk of store-bought self-test kits
Researchers from the University of Birmingham have warned that high street self-tests for conditions like vitamin deficiencies, menopause, and bowel cancer may not be fit for purpose. A review of 30 self-tests found many lacked crucial information on who should use them, how to interpret results, or what actions to take afterwards. Concerns were raised about the accuracy claims of these tests, with evidence often being unavailable or of low quality, and 18 tests were deemed "high risk". Experts, including Professor Kristien Boelaert, highlighted the potential for "false reassurance" and increased demand on the NHS due to these unregulated products. Calls have been made for urgent, tighter regulation of the booming self-test market, which is projected to reach £660m by 2030 in the UK.
BBC News
19 minutes ago
- BBC News
New cancer pain relief procedure becomes available in Leeds
A new procedure to reduce pain for cancer patients has been carried out in Yorkshire for the first time. Leslie Thompson, 79, was the first person in the region to undergo the treatment, at St James' Hospital in procedure, which is being offered to some terminally ill cancer patients, involves heating up nerves at the top of the spinal cord to permanently relieve the pain on the affected side of the body. Mr Thompson, from Yeadon, said he thought the treatment was a "good idea", having suffered "unbelievable" pain from lung cancer. Leeds has become the fourth centre in the UK to offer the procedure, after London, Glasgow and Liverpool. NHS bosses said eligible patients from across Yorkshire will no longer have to travel as far for the after the procedure, Mr Thompson said: "I feel less pain in my lung. I think it's a good idea and it will help a lot of other people. "The pain you get with cancer is unbelievable and it affects lots of people." While the nerves responsible for causing the pain are burnt in the neck, patients still retain their ability to walk and move around afterwards. The procedure involves using a radiofrequency generator, and needles which heat up and burn the nerves on the opposite side of the neck to where the pain is treatment is only suitable for patients who have pain on one side of the body and are well enough to benefit from the procedure, according to the NHS. Dr Sheila Black, consultant in anaesthesia and pain management at Leeds Teaching Hospitals NHS Trust, said the procedure would make the last weeks and months of a patient's life "more comfortable". She said: "I asked Leslie after the procedure: "How is your pain?" and he said: "What pain?". That's really wonderful to hear."Lots of the other treatments we have for pain have side effects, like morphine, which can cause sickness and constipation and sometimes still not help relieve the pain." Listen to highlights from West Yorkshire on BBC Sounds, catch up with the latest episode of Look North.
Daily Mirror
an hour ago
- Daily Mirror
People born between 2000 and 2009 urged to get lifesaving jab
Experts have expressed concern as uptake of the vaccine has declined People born between certain years are being urged to come forward for a 'lifesaving' vaccine. The NHS is appealing to hundreds of thousands of young people who have yet to receive the crucial human papillomavirus (HPV) vaccine. This jab can protect you against cervical and other forms of cancer. GP surgeries throughout England will be dispatching vaccine invitations to patients born between 2000 and 2009 (meaning they are aged 16 to 25) whose records indicate they missed their HPV vaccination at school. The vaccine is routinely administered annually to boys and girls in year 8 (aged 12 to 13) to safeguard them from contracting the HPV infection - which is responsible for nearly all cervical cancers and is associated with cancers of the mouth, throat, anus, penis and vagina. If anyone misses the jab, NHS teams conduct school catch-up visits and organise community clinics for home-schooled children. However, over the past three years alone, more than 418,000 children left school without receiving the HPV vaccination. It's believed there are many others now aged 16 to 25 who were not vaccinated at school. Therefore, NHS leaders aim to reach as many of these individuals as possible. That is in line with plans to eradicate cervical cancer by 2040 as outlined in the recently published 10 Year Health Plan. The NHS intends to increase uptake among girls to 90 per cent by 2040, whilst also boosting the number of women undergoing cervical screening. During the 2023/24 academic year, vaccination rates for 14 to 15-year-olds reached 76.7 per cent for girls and 71.2 per cent for boys by year 10. There was also a rise in uptake among year 8 pupils compared to the previous year, with 72.9 per cent of girls and 67.7 per cent of boys receiving the vaccine in 2023/24, up from 71.3 per cent and 65.2 per cent respectively for the first dose in 2022/23. Prior to 2023, two doses were necessary. Dr Amanda Doyle, national director of primary care and community services, said: "This vaccine is hugely important in our fight to eradicate cervical cancer but it isn't just for girls and women – it's also vital for boys and men to get vaccinated to protect themselves against cancers of the mouth, throat or genitals, while preventing spreading the HPV infection to sexual partners who could then develop cervical cancer. "Too many lives are lost to cervical cancer so the hard work of NHS staff across the country in vaccinating and screening as many people as possible will help us to meet our ambition of wiping out this disease. Encouraging progress has been made recently in increasing uptake but we know there is much more to do. "So if you're eligible for a HPV vaccination or are the parent of a child who is eligible, but didn't get the vaccine at school when they had the chance, I would urge you to come forward when your GP contacts you. Only a single dose is required and it could save you or your child's life." Public Health and Prevention Minister Ashley Dalton added: "The HPV vaccine is our most powerful tool in our fight to eliminate cervical cancer by 2040. But we've got a long way to go. Thousands of young people are still unvaccinated and vulnerable to this disease – which is so easily prevented. "If you missed your vaccination at school, it's not too late. Don't hesitate to make an appointment with your GP. One jab could save your life." The vaccine is also available to people up to the age of 45 with compromised immune systems and men who engage in sexual activity with other men. The most recent HPV vaccine, launched in England in 2021, has demonstrated increased effectiveness. It's projected to decrease instances of women's cancer by 16 per cent and HPV-related fatalities by nine per cent, compared to its predecessor. Research conducted in England indicates that the HPV vaccine prevents 90 per cent of cervical cancer cases. Just last month, the NHS initiated a new 'ping and book' service, sending digital invitations and reminders for cervical screenings via the NHS App.
