Bloomberg Businessweek Highlights Promise of Potential Drug
VX-770

March 22, 2011 

A new CF treatment known as VX-770 will be a groundbreaking medicine if approved by the U.S. Food and Drug Administration, says Bloomberg Businessweek in this week’s edition.

According to the article, VX-770 will be first pill designed to repair a genetic flaw based on the discovery of a mutation that causes a disease.

VX-770 is in the third and final stage of testing required for FDA approval. The drug was discovered in collaboration with the CF Foundation, which invested about $75 million in the project, and is being developed by Vertex Pharmaceuticals.

In a study of VX-770 released in February, the drug increased lung function by 10.5 percent over a year, reduced patients' need for antibiotics, and led to an average weight gain of seven pounds, the article notes. "We were euphoric," recalls Robert Beall, chief executive officer of the CF Foundation.

VX-770 is designed for a mutation, G551D, carried by about four percent of people with CF.

The CF gene was discovered in 1989. The news story describes the efforts of the CF Foundation, following the gene discovery, to encourage drugmakers to harness cutting-edge technology to develop new therapies to fight CF.

The magazine quotes Francis Collins, director of the National Institutes of Health and co-discoverer of the CF gene: “Discovering the genetic cause of a disease can lead you toward a therapy you never would have thought of otherwise."

Read the full Businessweek story on VX-770.