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Vertex Releases Promising Phase 2 Results of Ivacaftor in Residual Function Mutations of CF

June 4, 2014

Vertex Pharmaceuticals Inc. today announced preliminary results from a Phase 2 proof-of-concept study of ivacaftor (Kalydeco™) in people with cystic fibrosis who have what are known as “residual function” mutations of CF. The results showed improvements in lung function after two weeks of treatment. Vertex plans to initiate a larger Phase 3 trial to study the drug over a longer period of time in people with residual function mutations, pending discussions with regulatory authorities.

“These promising results are an important part of ongoing efforts to bring effective treatments to all people with cystic fibrosis,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. 

Read the Vertex press release.

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