NICE appraisal of the vanzacaftor triple therapy

The vanzacaftor triple therapy is a new, once-daily modulator therapy made by Vertex Pharmaceuticals. It is made up of tezacaftor, one of the modulators used in Kaftrio, as well as two new modulators called vanzacaftor and deutivacaftor.  

In December 2024, the US medicine regulator – the Food and Drug Administration – approved the vanzacaftor triple therapy for people with CF aged 6 and older who have one or more F508del mutations. In the US, the vanzacaftor triple therapy is called Alyftrek, but it could have a different name in the UK.  

Vertex have applied to the Medicines and Healthcare products Regulatory Agency (MHRA), the UK’s medicine regulator who are responsible for ensuring medicines are safe. We expect that the MHRA will soon respond to Vertex’s application.  

The vanzacaftor triple therapy will also be assessed by the National Institute for Health and Care Excellence (NICE), to determine its clinical and cost-effectiveness. This is separate to the recent appraisal of Kaftrio, Symkevi and Orkambi which concluded in 2024. This assessment will be more streamlined than the appraisal of Kaftrio, Symkevi and Orkambi, and NICE are expecting this process to conclude in summer 2025.   

As part of this NICE appraisal, Cystic Fibrosis Trust has submitted evidence to NICE, highlighting: 

• Our previous submissions to NICE which demonstrated what it is like to live with CF,  

• The importance of treatment choice, particularly for people with CF who cannot benefit from current CFTR modulators – either because of eligibility or side effects 

• The potential reduction in treatment burden with a once-daily CFTR modulator.  

As this is a more streamlined appraisal than the assessment of Kaftrio, Orkambi and Symkevi, we don’t yet know what further engagement there will be, but we will keep the CF community updated throughout. Please be reassured that access to Kaftrio will continue as usual, and nobody will be switched if they don't want to be. 

Please get in touch with our Helpline with any questions. You can call us on 0300 373 1000 or 020 3795 2184, email [email protected], or message us on WhatsApp on 07361 582053.  The Helpline is open Monday to Friday, 10am–4pm. 

You can read more about the vanza triple in our Q&A