VX-770, A Promising CF Therapy that Addresses Basic Defect, Enters Pivotal Clinical Trials

May 27, 2009

Vertex Pharmaceuticals Incorporated announced today the initiation of a Phase 3 registration program for VX-770, a promising oral drug aimed at treating the underlying cause of cystic fibrosis (CF).

This registration program is designed to generate data that the U.S. Food and Drug Administration can use to determine if VX-770 is safe, effective and acceptable for approval. The VX-770 registration program will consist of three different clinical trials, including: 

  • A primary 48-week trial for patients age 12 years and older who carry a G551D mutation of CF; 
  •  A 48-week trial for patients ages 6 to 11 years who carry a G551D mutation of CF; and 
  •  A 16-week trial to evaluate for the first time patients age 12 years and older who are homozygous for the Delta F508 mutation of CF, the most common mutation in CF patients. 

“The initiation of the VX-770 registration program is a major advancement in our efforts to bring forward a new therapy aimed at treating the underlying cause of CF,” said Robert J. Beall, Ph.D., president and chief executive officer of the Cystic Fibrosis Foundation. “This investigational drug represents one of the most promising routes to changing the course of this disease, and we are encouraged with the progress of both VX-770 and VX-809, which recently entered a Phase 2a clinical trial in CF patients.”

VX-770 is being developed and tested by Vertex, with support from the CF Foundation. The potential drug aims to open the chloride channels that do not function.

For more information about these trials, call the clinical trials hotline operated by Vertex at (877) 634-8789.

Read the Vertex Pharmaceuticals Inc. News Release.

Learn more about VX-770 and VX-809.