NICE recommends innovative cystic fibrosis therapy

National Institute for Health and Care Excellence (NICE) has recommended Alyftrek^® (deutivacaftor/tezacaftor/vanzacaftor) for eligible NHS patients with cystic fibrosis. The treatment was licensed for UK use in March.

Individuals who are six years old and over with at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene can access Alyftrek through NHS England.

NICE’s decision about the once-daily triple therapy comes one year after its approval of Kaftrio^®. The European Commission granted an expanded indication for Kaftrio in combination with ivacaftor in 2023.

NICE’s present recommendation of Alyftrek follows its comparative assessment between Alyftrek and Kaftrio. Evidence demonstrated that the CFTR modulator is as effective as Kaftrio, with similar costs.

Improving treatment for cystic fibrosis

“CFTR modulators are already revolutionising the way cystic fibrosis is treated so we’re pleased to be able to recommend Alyftrek, the latest of this type of treatment that has been shown to be effective, with significant benefits for people with the condition,” stated Helen Knight, Director of Medicines Evaluation at NICE.

Alyftrek addresses the underlying cause of cystic fibrosis instead of simply managing symptoms, according to NICE.

Availability of Alyftrek on the NHS “represents a significant milestone in our journey to serially innovate and further improve the lives of people living with this disease,” Ludovic Fenaux, Senior Vice President, Vertex International stated. “In our pivotal studies, Alyftrek^® demonstrated the potential for even better outcomes for patients than Kaftrio (ivacaftor/tezacaftor/elexacaftor)”.

Following the European regulatory approval, eligible individuals in Ireland, Denmark and Germany will be the first to access deutivacaftor/tezacaftor/vanzacaftor in the EU. NICE stated that the indication represents approximately 89 percent of cystic fibrosis patients in England.

NICE’s final draft guidance for this medicine is expected to be published on 30 July.