Positive Results for Phase 3 Studies of the Tezacaftor/Ivacaftor Combination Treatment

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced that the New England Journal of Medicine (NEJM) published two articles with results from two Phase 3 studies of the tezacaftor/ivacaftor combination treatment, a medicine in development that is designed 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 F508delmutation, 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 today in conjunction with two oral presentations at the 31st Annual North American Cystic Fibrosis Conference, November 2 to 4, 2017 in Indianapolis. 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.

For more in-depth results and summaries for both the EVOLVE and EXPAND studies, visit the originally published article at: http://www.businesswire.com/news/home/20171103005529/en/

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