Vertex has announced successful results from two Phase III studies of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of certain cystic fibrosis (CF) in people ages 12 years and older.
The results show that both studies met the primary and all key secondary endpoints, demonstrating significant improvements in lung function and other measures of the disease.
The data, published in The Lancet and The New England Journal of Medicine (NEJM), showed that the drug was generally well tolerated in patients with at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation.
The results are “impressive and represent a historic moment in CF care, with the medicine demonstrating improvements in multiple CF outcome measures in clinical trials, while being generally well tolerated,” commented Raksha Jain, associate professor, internal medicine, University of Texas Southwestern Medical Center and lead author of the NEJM publication.
Trikafta recently won US Food and Drug Administration (FDA) approval for the treatment of CF, meaning that 6,000 potential patients will be able to receive the treatment, and a further 12,000 who are currently eligible for one of Vertex’s three other FDA-approved CF medicines are now also eligible for Trikafta.
Vertex has also submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the treatment, and is currently evaluating it in people ages six through 11 with F/MF and F/F CF mutations in an ongoing Phase III study.
The company also recently signed a definitive agreement with NHS England to make all three of their UK-licensed cystic fibrosis medicines available - Orkambi (ivacaftor/lumacaftor), Symkevi (tezacaftor/ivacaftor) and Kalydeco (ivacaftor).