Phase III studies of VX 661+ Kalydeco in cystic fibrosis published in NEJM.- Vertex Pharma.

Vertex Pharmaceuticals announced that the New England Journal of Medicine (NEJM) published two articles with results from two Phase III studies of the VX 661+ Kalydeco (tezacaftor + ivacaftor) combination treatment, to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. In both studies, the tezacaftor/ivacaftor combination demonstrated statistically significant and clinically meaningful improvements in lung function and other measures of disease.

The EVOLVE study evaluated the combination in people who have two copies of the F508del mutation, the most common mutation in the CFTR gene. The EXPAND study evaluated the combination in people with one F508del mutation and one mutation that results in residual CFTR function.

The results were published in two online articles in conjunction with two oral presentations at the 31st Annual North American Cystic Fibrosis Conference. These data formed the basis of applications for the approval of the tezacaftor/ivacaftor combination that are currently under review with regulatory agencies in the United States and Europe. In the United States, the Food and Drug Administration (FDA) has granted Priority Review of the application and has set an action date of February 28, 2018.

See: Tezacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del