Vertex Pharmaceuticals (VRTX +3%) perks up on light volume on the heels of its announcement that positive results from two Phase 3 clinical trials assessing the combination of tezacaftor/ivacaftor in cystic fibrosis (CF) patients were just published in the New England Journal of Medicine and also presented at the 31st Annual North American Cystic Fibrosis Conference in Indianapolis, IN.
The EVOLVE trial evaluated the combination in CF patients who carried two copies of the F508del mutation while the EXPAND study enrolled those with one F508del mutation and one mutation that results in residual CFTR function (mutations in the CFTR gene cause CF).
Both studies demonstrated statistically valid improvements in lung function with favorable safety profiles. EVOLVE also showed a significant reduction in pulmonary exacerbations and lower body fat.
The company's marketing applications in the U.S. and Europe are currently under review. The FDA's action date is February 28.
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