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Scientists comment on NICE final draft guidance and the NHS rollout of Alyftrek for Cystic Fibrosis.
Prof David Sheppard, Professor of Physiology, University of Bristol, said:
“It is very welcome news that Alyftrek will become available to people with cystic fibrosis in England. Alyftrek is the latest transformational drug therapy for cystic fibrosis, which works by targeting the root cause of the disease, genetic defects that disable or destroy the protein CFTR. CFTR forms a gated pathway for chloride, one part of salt to cross cell borders lining ducts and tubes throughout the body, such as the air passageways and bowel. Loss of the CFTR protein causes ducts and tubes to become blocked by sticky mucus.
“Alyftrek is a tablet taken by mouth, which consists of three drugs vanzacaftor, tezacaftor and deutivacaftor. Vanzacaftor and tezacaftor allow defective CFTR proteins trapped inside cells to reach the cell border. Like oil on a rusty gate, deutivacaftor improves the activity of CFTR proteins at the cell border.
“When compared with the current triple combination therapy, Kaftrio (elexacaftor-tezacaftor-ivacaftor), Alyftrek has the important advantage of being a once daily tablet taken by mouth rather than a twice daily tablet taken by mouth. In clinical trials, Alyftrek caused greater reductions in the saltiness of sweat (a hallmark of cystic fibrosis) than Kaftrio, suggesting that Alyftrek restores greater CFTR function than Kaftrio.
“Alyftrek is being made available to people with cystic fibrosis and one or two copies of the most common cause of cystic fibrosis, the F508del defect. In the UK, about 90% of people with cystic fibrosis will likely benefit from access to Alyftrek. However, the United States drug regulator, the FDA, has allowed access to Alyftrek for people with cystic fibrosis and additional genetic defects, totally around 300 rare defects in the CFTR gene. It is therefore highly likely that additional people with cystic fibrosis in England would likely benefit from access to Alyftrek. I urge that Alyftrek is make available to all people with cystic fibrosis and genetic defects responsive to the drug.”
David Ramsden, Cystic Fibrosis Trust Chief Executive, said:
“Today’s announcements are a positive step in the journey to better treatments for more people with cystic fibrosis – a lifelong, life-limiting condition without a cure. It’s thanks to the incredible hard work and support of the CF community and everyone we work with that modulator drugs are now a treatment option for many, but not all people with CF.
“Cystic fibrosis continues to make lives too tough and too short, which is why we will continue funding vital research to work towards a future where everyone can benefit from a life unlimited by CF.”
Prof Kevin Southern, Professor of Child Health, Chair, UK CF Medical Association, said:
“The CFMA welcome the approval of Alyfrek, which may offer an important alternative for some people with CF who are already treated with Kaftrio, or be an option for a small number of people who are not eligible for current therapies or have not been able to tolerate them. CF remains a condition without a cure and while improvements to therapies is good news, ongoing challenges exist for people with the condition. CF clinical teams will continue to work to achieve excellent health outcomes and quality of life for all, and we hope that in the future, new therapies will become available for people with CF, particularly for those who do not respond to existing therapies.”
The NICE final draft guidance on Alyftrek was published on the NICE website at 00:01 UK time on Tuesday 15 July.
https://www.nice.org.uk/guidance/indevelopment/gid-ta11430/documents
Declared interests
David Ramsden: No COI
Kevin Southern: No COI.
Prof David Sheppard: Currently, research grants from the Cystic Fibrosis Trust.
Previously, a research grant from Vertex Pharmaceuticals through the company’s Vertex Innovation Awards programme.