Vertex Pharmaceuticals’ triple combination cystic fibrosis (CF) therapy has been recommended by the National Institute for Health and Care Excellence (NICE) to treat patients aged six years and older.
The health technology assessment agency has recommended in final draft guidance that the once-daily drug, Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), be used on the NHS in England to treat CF patients who have at least one F508del mutation or another responsive mutation in the CF transmembrane conductance regulator (CFTR) gene.
Approximately 11,000 people in the UK are affected by CF, a rare inherited disease caused by a faulty CFTR gene, which helps regulate the flow of water and chloride into and out of the cells in a number of organs.
In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic infections and progressive damage that eventually leads to death for many patients.
Vertex’s Alyftrek is a CFTR modulator that works by correcting the malfunctioning protein made by the CFTR gene, helping patients live more independently and reducing the time they need to spend in hospital.
The drug will now be immediately funded by the NHS in England.
Helen Knight, director of medicines evaluation at NICE, said: “CFTR modulators are already revolutionising the way CF 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.”
NICE’s decision, which follows the Medicines and Healthcare products Regulatory Agency’s approval of the drug in March, was supported by clinical evidence showing that Alyftrek was as effective at improving lung function as Vertex’s currently approved triple combination therapy Kaftrio (ivacaftor/tezacaftor/elexacaftor), and more effective at reducing sweat chloride levels.
Ludovic Fenaux, senior vice president, Vertex International, said: “In our pivotal studies, Alyftrek demonstrated the potential for even better outcomes for patients than Kaftrio.
“We’re pleased to have reached this agreement with NHS England that recognises the value that this new medicine brings to CF patients, their families and society.”
