Hundreds of cystic fibrosis patients become eligible for ‘life-changing' therapy

Rhyl Journal

2 days ago

The National Institute for Health and Care Excellence (Nice) has given the green light for the NHS to give patients Alyftrek, a type of modulator therapy which works to tackle the underlying cause of cystic fibrosis (CF).
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'.