A new once-daily cystic fibrosis (CF) treatment trialled in Manchester has been approved for NHS use, following a recommendation made earlier this month by the National Institute for Health and Care Excellence (NICE).
Clinical trials delivered at the National Institute for Health and Care Research (NIHR) Manchester Clinical Research Facility (CRF) have shown the new triple therapy to be at least as effective as current drug Kaftrio® at improving lung function for people aged over 12 with CF caused by a specific gene (F508del) mutation.
The NIHR Manchester CRF at Wythenshawe Hospital, was the top-recruiting UK site for the adult study for the modulator therapy Vanzacaftor–tezacaftor–deutivacaftor, also known as Alyftrek®, which helps target the underlying cause of the disease.
Professor Alex Horsley, Medical Director of the NIHR Manchester CRF at Wythenshawe Hospital, was the Lead Investigator. He said: “What we have seen in this study is that this new triple combination therapy is as good for lung function. We have also seen a slightly bigger impact on sweat chloride levels, which means it likely has a better effect upon the core problem in CF.
“As the treatment is taken just once daily, this could also make a big difference to people with CF, with more convenience and flexibility, helping simplify their treatment regimens, which are already very complicated.”
Cystic Fibrosis is a rare genetic condition affecting more than 10,000 people in the UK. It causes thick, sticky mucus to build up in the lungs, digestive system and other organs causing a wide range of challenging symptoms.
The condition is caused by reduced quantity and/or function of the CF transmembrane conductance regulator (CFTR) protein that regulates levels of sodium and chloride in cells. A person will be born with CF when the faulty CFTR gene from both parents is genetically passed on.
