BETHESDA, Md., June 10, 2011 /PRNewswire-USNewswire/ -- Vertex Pharmaceuticals Inc. announced a plan to provide VX-770, a CF medicine in development, to people with the G551D mutation who are in critical medical need and may benefit from treatment prior to potential approval of the drug from the U.S. Food and Drug Administration (FDA).
Pending FDA review and approval, Vertex expects to open the program at clinical sites in the United States as early as July.
"We are delighted that Vertex is making VX-770 available to patients through an expanded access program," said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. "This underscores Vertex's commitment to do what is right for the CF community."
The company is on track to submit a New Drug Application for VX-770 to the FDA in the second half of 2011.
VX-770 is an oral drug in development that targets the underlying cause of cystic fibrosis.
Results from Phase 3 clinical trials released this year showed that those who took the drug had marked improvements in lung function and other key indicators of the disease, including sweat chloride levels, likelihood of pulmonary exacerbations and body weight.
The CF Foundation worked with Vertex to discover VX-770, and has provided substantial scientific, financial and clinical support throughout the development process.
The Vertex VX-770 drug access program is for people with highly limited lung function and who meet other criteria. For information on eligibility or other details, call Vertex Medical Information at 1-877-634-VRTX (8789).
About the Cystic Fibrosis Foundation
The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization. For more information, go to www.cff.org.
SOURCE Cystic Fibrosis Foundation