Latest news with #CFTR
Medscape
17 hours ago
- Health
- Medscape
NHS to Offer New Cystic Fibrosis Therapy Alyftrek
Hundreds of people with the most common type of cystic fibrosis could be offered a new triple therapy after the National Institute for Health and Care Excellence (NICE) recommended vanzacaftor–tezacaftor–deutivacaftor (Vnz–Tez–Diva) for routine NHS use in England. The once-daily oral treatment, branded as Alyftrek and manufactured by Vertex, is aimed at individuals aged 6 and over who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. When this gene is faulty, cells are unable to make normal CFTR protein to regulate intracellular levels of sodium and chloride. This leads to a buildup of the thick, sticky mucus characteristic of cystic fibrosis. Modulators Target Underlying Cause Vanzacaftor and tezacaftor are CFTR correctors, binding to different sites on the CFTR protein to increase its presence on cell surfaces. Deutivacaftor enhances the activity of the defective CFTR protein at the cell surface. These combined actions reduce the thickness of mucus in the lungs and digestive system. In final draft guidance, NICE said its recommendation formed part of "an evolving treatment landscape for cystic fibrosis", in which the advent of gene modulators is shifting care away from symptom management towards targeting the underlying cause of the disease. Helen Knight, director of medicines evaluation at NICE, said that CFTR modulators were "revolutionising" the way that cystic fibrosis is treated. An Alternative to Kaftrio Alyftrek was licensed in the UK in March this year and approved for use by the European Medicines Agency (EMA) in April. Alyftrek was previously designated an orphan medicine by the EMA. NICE's recommendation follows a rapid assessment that compared Alyftrek with the company's other NICE-approved triple therapy, Kaftrio (elexacaftor-tezacaftor-ivacaftor, Vertex). Evidence suggests Alyftrek is as effective as Kaftrio in improving lung function, growth, and weight gain, and in reducing the number of lung infections. Alyftrek has the additional advantage of once-daily dosing, compared with Kaftrio's twice-daily regimen. NICE said that Alyftrek could be offered to patients who are not eligible or able to take Kaftrio, as well as an alternative for those already taking Kaftrio. Alyftrek is available in two dose formulations: Vnz 10 mg / Tez 50 mg / Diva 125 mg (84-tablet pack) Vnz 4 mg / Tez 20 mg / Diva 50 mg (56-tablet pack) Both pack sizes have a list price of £16,110, excluding VAT. NICE's recommendation is dependent on the company providing the drug under the terms of a confidential commercial arrangement. Clinicians are advised to prescribe the least costly suitable treatment, factoring in dosage, administration costs, and commercial terms. David Ramsden, chief executive of the Cystic Fibrosis Trust, called the decision "a positive step in the journey to better treatments for more people with cystic fibrosis". Similar announcements are expected to follow in Scotland, Wales, and Northern Ireland, the trust said. NHS Expands Access Beyond NICE Scope Following the NICE recommendation, NHS England (NHSE) announced that it had secured a commercial deal with Vertex to expand access to Alyftrek beyond the patient group considered in the regulator's evaluation. As a result, 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, it said. NHSE estimated that around 95% of people with cystic fibrosis in England are now eligible for modulator therapy. John Stewart, NHSE's director for specialised commissioning, described the move as 'a major leap forward'. '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," he said. Stewart added that once-daily treatment at home could reduce hospital visits and help children live more freely and independently.
Business Wire
19 hours ago
- Health
- Business Wire
Cystic Fibrosis Foundation Commits Up to an Additional $24 Million for Prime Medicine to Develop Gene Editing Therapy
BETHESDA, Md.--(BUSINESS WIRE)--Today, the Cystic Fibrosis Foundation announced an additional investment of up to $24 million in Prime Medicine to continue the development of a gene editing therapy for people with cystic fibrosis (CF). Prime Medicine uses a gene editing technology called prime editing — a technology that enables a wide range of modifications to the DNA with a high degree of precision. The company — founded by Drs. David Liu and Andrew Anzalone, who pioneered the development of this unique editing technology — is investigating whether prime editing could treat several diseases, including CF. The CF Foundation's initial investment in Prime Medicine supported work to demonstrate the versatility of prime editing to correct multiple CF-causing mutations in the lab. This additional investment by the CF Foundation will focus on the development of a prime editing therapy targeting the nonsense mutation G542X — one of the most prevalent CF-causing nonsense mutations and one for which there are no available therapies. 'We believe gene editing offers the best hope for a cure for cystic fibrosis because it could permanently correct the mutations that cause this disease,' said Michael P. Boyle, MD, president and chief executive officer of the Cystic Fibrosis Foundation. 'Our investment will help support gene editing research for CF through early development stages, including efforts to overcome the challenges of delivery to the lungs.' As part of its ongoing efforts to develop a prime editing therapy, Prime Medicine is investigating the best way to deliver its gene editing treatment to the lungs of people with CF, a major challenge because of the thick, sticky mucus that clogs the airways and the body's natural immune response to block foreign entities from entering cells. If successful, the gene editing cargo will enter the lung cells that normally make CFTR protein and correct the CFTR mutation in the cell's DNA, resulting in a potentially permanent fix to the CFTR gene. With continued advancement in prime editing and delivery to the lungs, this technology may eventually be used to correct many types of CF mutations. Prime Medicine is advancing multiple approaches in CF, including 'hotspot,' which uses prime editing to make smaller corrections to specific CFTR mutations and PASSIGE, which uses prime editing to make large gene insertions. These combined strategies eliminate the need to develop new prime editing therapies for each individual mutation. Previously, the Foundation committed up to $15 million to Prime Medicine to pursue development of their gene editing technology for CF. About the Cystic Fibrosis Foundation The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, visit
Leader Live
2 days ago
- Health
- Leader Live
Hundreds of cystic fibrosis patients become eligible for ‘life-changing' therapy
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'.
South Wales Guardian
2 days ago
- Health
- South Wales Guardian
Hundreds of cystic fibrosis patients become eligible for ‘life-changing' therapy
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'.
Rhyl Journal
2 days ago
- Health
- Rhyl Journal
Hundreds of cystic fibrosis patients become eligible for ‘life-changing' therapy
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'.
