US biotech Vertex has revealed positive phase III results of its tezacaftor/ivacaftor treatment for the underlying cause of cystic fibrosis (CF).
The therapy for patients aged 12 and over targets certain mutations in the cystic fibrosis transmembrane conductance regulatory (CFTR) gene, specifically patients who have the F508del mutation.
Results from the phase II EVOLVE study evaluated people with two copies of the F508del mutation – the most common mutation of the CFTR gene.
The 24-week study met its primary endpoint with a 4% increase in the mean absolute improvement in lung function. Those receiving the combination also saw a 35% reduction in the annualised rate of pulmonary exacerbations compared to those on placebo.
Those treated with tezacaftor/ivacaftor also saw improvements across multiple disease measures, such as an improved lung disease, which is the leading cause of death in people with CF.
A second study known as EXPAND evaluated patients with just one F508del mutation over eight weeks of treatment. It too met its primary endpoint, with a 6.8% increase in absolute improvement in lung function.
Jeffrey Chodakewitz, executive vice president and chief medical officer, Vertex, said: “We have made unprecedented progress in the treatment of CF in recent years, but we continue to drive ourselves to deliver even greater benefits for patients today and more new medicines for patients who are still waiting.
“These tezacaftor/ivacaftor results are exciting because they represent the potential to do both.”
The combination therapy is currently under review in both Europe and the US, where it was granted priority review from the Food and Drug Administration (FDA).