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New England Journal of Medicine Features Study of Kalydeco
(VX-770) – First Potential Therapy Targeting Underlying Cause of Cystic Fibrosis

November 2, 2011

The New England Journal of Medicine, the world’s most widely read and influential medical periodical, features a study this week about a Phase 3 clinical trial of Kalydeco™ (VX-770), a potential CF therapy undergoing review for approval by the U.S. Food and Drug Administration (FDA).

If approved, Kalydeco will be the first drug on the market that targets the underlying cause of cystic fibrosis — a faulty gene and its protein product, CFTR — rather than just the symptoms of the disease.

The maker of Kalydeco, Vertex Pharmaceuticals, Inc., is seeking approval for the drug in people ages 6 and older with at least one copy of the G551D mutation of CF.

Kalydeco was developed by Vertex with CF Foundation support and research input.

The VX-770 study is “a great victory in the war against genetic diseases and marks the end of the beginning for the treatment of the cystic fibrosis defect,” writes Pamela B. Davis, M.D., Ph.D., dean of the Case Western Reserve University School of Medicine, in an independent editorial accompanying the Journal study.

The article presents results from a Phase 3 clinical trial of Kalydeco in people ages 12 and older with the G551D mutation of CF. People who took the drug showed dramatic improvements in lung function and other key symptoms of CF, compared with those who did not receive Kalydeco, and they maintained these improvements throughout the nearly yearlong trial.

The New England Journal of Medicine study marks an important and exciting milestone for the cystic fibrosis community, and it confirms that we are one step closer to finding a cure for the disease,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “With Kalydeco on track for possible FDA approval in 2012, we are moving forward energetically to identify and develop additional therapies that target the underlying cause of the disease to treat all people with CF.”

Kalydeco is currently being evaluated in combination with another oral drug in development, VX-809, in people with at least one copy of the most common mutation of CF, Delta F508. Vertex is enrolling participants for the second part of an ongoing Phase 2 study of the two drugs.

In 2012, Vertex also plans to begin a Phase 2 clinical trial of Kalydeco in combination with another potential therapy, VX-661, in people with two copies of the Delta F508 mutation of CF.

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