Phase 3 Study of VX-770 in Children Shows Marked Improvement in Lung Function and Other Measures

March 29, 2011

Vertex Pharmaceuticals and the Cystic Fibrosis Foundation today announced positive results from an ongoing Phase 3 clinical trial of VX-770 for children age 6 to 11.

VX-770 is an oral drug in development that targets the underlying cause of cystic fibrosis.

Children who were on the drug showed a marked improvement in lung function through 24 weeks of a 48-week trial, compared to those who took the placebo.

The trial is designed to evaluate patients who carry at least one copy of a CF mutation called G551D and included 52 children.

Patients in the trial also showed improvements in secondary endpoints of the study, including weight gain and sweat chloride levels -- areas critical to the health of people with CF.

VX-770 is being developed by Vertex Pharmaceuticals and was discovered in collaboration with the CF Foundation.

“The findings announced today are highly encouraging and reinforce the concept that we can significantly improve clinical outcomes for CF patients by repairing the defective protein that causes the disease,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “We are hopeful that by continuing on this path, we will ultimately have a profound impact on all who suffer from cystic fibrosis.”

The new data mirrors promising results announced in February 2011 of a Phase 3 trial of VX-770 in people with CF, ages 12 and older. In that trial, patients who received VX-770 showed marked improvements in lung function and other key clinical indicators of cystic fibrosis. 

Vertex plans to submit a New Drug Application for VX-770 to the U.S. Food and Drug Administration in the second half of 2011. Generally, the FDA takes between 6 and 12 months to review and rule on a drug application. 

About 4 percent of people with CF carry the G551D mutation. More studies are needed to determine whether other CF mutations may benefit from VX-770.

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