Vertex Phase 3 Cystic Fibrosis Drug Trials Show Promising Results

Vertex Pharmaceuticals Incorporated has announced results from two Phase 3 studies of the tezacaftor/ivacaftor combination treatment that showed statistically significant improvements in lung function in people with cystic fibrosis (CF) ages 12 and older who have certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The 24-week EVOLVE study evaluated the combination treatment in people who have two copies of the F508del mutation. The second study, EXPAND, was an 8-week crossover study that evaluated the combination treatment in people who have one mutation that results in residual CFTR function and one F508del mutation. Both studies met their primary endpoints. Based on these results, Vertex plans to submit a New Drug Application (NDA) to the FDA and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the third quarter of 2017 for the tezacaftor/ivacaftor combination treatment.

“The tezacaftor/ivacaftor combination treatment demonstrated clinically meaningful benefits, with a favorable safety profile, across multiple patient groups,” said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex. “This combination treatment may provide a promising new option for treating the underlying cause of CF in the future and brings us increasingly closer to our goal of developing new medicines for all people with the disease.” (Source: Vertex Website, 28 March, 2017)

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