Alyftrek approved by NICE and NHS England extends access to triple therapies

Alyftrek (deutivacaftor/tezacaftor/vanzacaftor), is a new once-a-day triple combination modulator drug. It provides a treatment option for some people who are not eligible or able to take Kaftrio and an alternative for people who are already taking Kaftrio. 

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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 roll-out 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.”

Catherine, whose daughter Kate, 11, has CF, said: “Although Kate had had a great response to Kafrio, we would expect her to have a better response to Alyftrek because Kate's second, rare mutation, is listed as responding to Alyftrek, whereas it does not respond to Kaftrio.

“Kaftrio completely changed the trajectory that Kate was on with her CF. It's fair to say that times were getting a little scary and CF was really starting to dominate our lives. She had a wonderful 18 months of staying well with minimal courses of oral antibiotics, although still more than most of her peers with CF, but eventually needed IVs and oral antibiotics again.

“We aren't expecting miracles, and it isn't lost on us how lucky we are to have Kaftrio, but we still certainly would take the opportunity to make things even better if we can. We are hoping Alyftrek will reduce the need for oral antibiotics and even better still, IVs. An added bonus is that it is a once a day dose. Although this doesn't seem like a huge thing, it is actually quite a pain having to have a high-fat snack in the evening. People don't realise what a burden CF is, modulators make things a lot better if you are lucky enough to be able to have them and tolerate them, but CF is still always there.”