NICE Recommends Vanzacaftor-Tezacaftor-Deutivacaftor for Cystic Fibrosis Treatment in England
核心洞察
NICE has approved vanzacaftor-tezacaftor-deutivacaftor (搜索) (Alyftrek) for NHS funding in England to treat cystic fibrosis in patients aged 6 years and older with at least one F508del (搜索) mutation.
Clinical trial evidence demonstrates the triple combination therapy is as effective as the current standard treatment ivacaftor-tezacaftor-elexacaftor (搜索) in improving lung function, growth, weight gain, and reducing lung infections.
The new therapy must be funded within 30 days of final guidance publication, with cost comparisons suggesting similar expenses to existing treatments.
The National Institute for Health and Care Excellence (NICE) has recommended vanzacaftor-tezacaftor-deutivacaftor (搜索) (Vnz-Tez-Diva; Alyftrek, Vertex) for NHS funding in England to treat cystic fibrosis in patients aged 6 years and older who have at least one F508del (搜索) mutation in the CFTR (搜索) gene. The decision mandates NHS funding within 30 days of final guidance publication.
Clinical Evidence and Efficacy
Clinical trial evidence demonstrates that Vnz-Tez-Diva performs as effectively as the current standard treatment ivacaftor-tezacaftor-elexacaftor (搜索) (Iva-Tez-Elx) in patients 12 years and over. The therapy showed comparable improvements in lung function, growth and weight gain, while reducing the number of lung infections. Although direct comparisons with Iva-Tez-Elx have not been conducted in patients aged 6 to 11 years, NICE concluded the treatment is likely to work equally well in this younger population.
Treatment Positioning and Licensing
The new triple combination therapy works through a similar mechanism to Iva-Tez-Elx, which currently serves as the usual treatment for cystic fibrosis patients with at least one F508del (搜索) mutation in the CFTR (搜索) gene. However, key licensing differences exist between the treatments. While Iva-Tez-Elx is licensed for patients 2 years and over, Vnz-Tez-Diva is approved for those 6 years and older.
Importantly, Iva-Tez-Elx is licensed specifically for people with at least one F508del (搜索) mutation and no other responsive mutation. In contrast, Vnz-Tez-Diva can be used in patients 6 years and over who have at least one F508del mutation or another responsive mutation in the CFTR (搜索) gene, potentially expanding treatment options for certain patient subgroups.
Cost Considerations and Access
A cost comparison between Vnz-Tez-Diva and Iva-Tez-Elx in patients 6 years and over suggests similar expenses. The list price for Vnz-Tez-Diva is £16,110.00 per 84-tablet pack of the higher-strength formulation (Vnz 10 mg, Tez 50 mg and Diva 125 mg) and the same price for a 56-tablet pack of the lower-strength tablets (Vnz 4 mg, Tez 20 mg and Diva 50 mg).
Vertex has established a commercial arrangement with NHS England (搜索) that provides the treatment with a discount, though the specific discount size remains commercially confidential. NICE determined there is sufficient evidence showing Vnz-Tez-Diva provides benefits and value for money, enabling routine use across the NHS for the approved patient population.
Implementation Requirements
Under NICE's recommendation, Vnz-Tez-Diva must be funded in the NHS in England for the specified condition and population if considered the most suitable treatment option. The guidance requires implementation within 30 days of final publication, and NICE has developed tools and resources to support healthcare providers in implementing this guidance across the health system.