New cystic fibrosis drug fast-tracked for NHS use
Some people with cystic fibrosis are set to benefit from a new once-daily triple therapy treatment, following NICE's recommendation of Alyftrek in final draft guidance published today.
The treatment will be available for people aged 6 years and over who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, representing approximately 89% of people with the condition in England.
Alyftrek (vanzacaftor–tezacaftor–deutivacaftor), made by Vertex, represents the latest advance in CFTR modulator treatments that are transforming cystic fibrosis care by targeting the underlying cause of the disease rather than just managing symptoms.
This recommendation broadens the number of people eligible for cystic fibrosis treatments
The recommendation follows a rapid assessment by NICE that compared Alyftrek with the company's other NICE-approved triple therapy, Kaftrio. Evidence suggests Alyftrek is as effective as Kaftrio, with similar costs, following a commercial agreement between NHS England and Vertex.
"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," said Helen Knight, director of medicines evaluation at NICE.
How the treatment works
Alyftrek combines three active components that work together to address the faulty CFTR protein. Vanzacaftor and tezacaftor act as CFTR correctors, binding to different sites on the CFTR protein to increase the amount present on cell surfaces. Deutivacaftor then improves the activity of the defective CFTR protein. These combined actions make lung mucus and digestive juices less thick, helping to relieve the debilitating symptoms of cystic fibrosis.
Cystic fibrosis is a progressive, life-limiting inherited condition affecting about 11,000 people in the UK. The faulty CFTR gene prevents cells from making functioning versions of a protein that regulates sodium and chloride levels, leading to a buildup of thick, sticky mucus in the body's tubes and passageways.
Licensed for use in the UK in March, Alyftrek will now be immediately funded by the NHS in England. This rapid timeline demonstrates NICE's commitment to getting breakthrough treatments to patients quickly while ensuring value for the NHS drugs budget.
The decision comes just over a year after NICE approved Kaftrio and highlights the evolving treatment landscape for cystic fibrosis, where new therapies are continuing to shift care towards addressing the root cause of the disease.