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December 2011

Drug Targeting Basic Defect Will Receive FDA Priority Review

Capri Faulk, a second-grader who took part in a Kalydeco clinical trial at East Tennessee Children’s Hospital in Knoxville, Tennessee.
Capri Faulk, a second-grader who took
part in a Kalydeco clinical trial at East
Tennessee Children’s Hospital in
Knoxville, Tenn.

Earlier this month, Vertex Pharmaceuticals, Inc., announced that the U.S. Food and Drug Administration (FDA) has granted a request for a six-month priority review of a potential new CF therapy, Kalydeco™, formerly known as VX-770.

If approved, Kalydeco (pronounced kuh-LYE-deh-koh) would be the first drug available that targets the underlying cause of CF.

Vertex is seeking approval of the drug for people ages 6 and older with the G551D mutation of CF.

The expedited review sets a target date of April 18, 2012, for the FDA’s approval decision, four months earlier than the standard review time of 10 months.

The FDA grants priority review status for several reasons — for example, if a potential drug is considered a major treatment advance.

Kalydeco was discovered in a collaboration between Vertex and the Cystic Fibrosis Foundation that began more than a decade ago, with the Foundation providing substantial scientific, financial and clinical support throughout the development process.

The FDA application follows the release earlier in 2011 of groundbreaking results from late-stage clinical trials of Kalydeco in people ages 6 and older with at least one copy of the G551D mutation of CF. Study volunteers who received the drug showed significant improvements in lung function, weight gain, pulmonary exacerbations and sweat chloride levels.

“This is truly an exciting time in the history of CF,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “We have seen that a drug targeting the underlying cause of CF can greatly improve the health and quality of life of people with the disease. This tremendous achievement will fuel our efforts as we focus on developing additional therapies to bring the hope offered by Kalydeco to all people with CF.”

In November, the influential New England Journal of Medicine published a study of a Phase 3 clinical trial of Kalydeco, which was described in an independent editorial as marking “the end of the beginning for the treatment of the cystic fibrosis defect.” The study attracted the attention of major national news organizations, including the Los Angeles Times, CNN and “ABC World News.”

Watch the video of one Kalydeco clinical trial participant, Lauren Brenneman, 33, an adult with CF and mother of 4-year-old Isaac, who also has CF.



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