Vanzacaftor-tezacaftor-deutivacaftor (Vnz-Tez-Diva) can be used as an option to treat cystic fibrosis in people 6 years and over who have at least 1 F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Vnz-Tez-Diva can only be used if the company provides it according to the commercial arrangement.
Use the least expensive option of the suitable treatments (including Vnz-Tez-Diva and ivacaftor-tezacaftor-elexacaftor [Iva-Tez-Elx]), having discussed the advantages and disadvantages of the available treatments with the person with the condition. Take account of administration costs, dosages, price per dose and commercial arrangements.
This recommendation is not intended to affect treatment with Vnz-Tez-Diva that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS healthcare professional consider it appropriate to stop. For children or young people, this decision should be made jointly by the healthcare professional, the child or young person, and their parents or carers.
What this means in practice
Vnz-Tez-Diva must be funded in the NHS in England for the condition and population in the recommendations, if it is considered the most suitable treatment option. Vnz-Tez-Diva must be funded in England within 30 days of final publication of this guidance.
There is enough evidence to show that Vnz-Tez-Diva provides benefits and value for money, so it can be used routinely across the NHS in this population.
NICE has produced tools and resources to support the implementation of this guidance.
Usual treatment for cystic fibrosis with at least 1 F508del mutation in the CFTR gene is Iva-Tez-Elx, which is licensed for people 2 years and over. Vnz-Tez-Diva works in a similar way and is licensed for people 6 years and over.
Clinical trial evidence shows that Vnz-Tez-Diva is as effective as Iva-Tez-Elx in people 12 years and over at improving lung function, growth and weight gain and reducing the number of lung infections. It has not been directly compared with Iva-Tez-Elx in people aged 6 to 11 years but is likely to work as well in these people.
A cost comparison of Vnz-Tez-Diva and Iva-Tez-Elx in people 6 years and over suggests that the costs are similar. But Iva-Tez-Elx is licensed for people with at least 1 F508del mutation and no other responsive mutation. So, Vnz-Tez-Diva can only be used in people 6 years and over who have at least 1 F508del mutation in the CFTR gene.
For all evidence see the committee papers. For more information on NICE's evaluation of Iva–Tez–Elx, see the committee discussion section in NICE's technology appraisal guidance on ivacaftor–tezacaftor–elexacaftor, tezacaftor–ivacaftor and lumacaftor–ivacaftor for treating cystic fibrosis.