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 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).
Statement from Vertex Pharmaceuticals Inc.:
VX-770 is a medicine in development for people with cystic fibrosis who are 6 years or older and have at least one copy of the G551D mutation. Vertex is on track to submit global regulatory applications for approval in the United States, Canada and Europe, including a New Drug Application (NDA) in the United States and a Marketing Authorization Application (MAA) in the European Union in the second half of 2011.
In recognition of the immediate needs of some people with CF, Vertex is planning an expanded access program for VX-770. This program is designed to provide VX-770 to people who have at least one copy of the G551D mutation who are in critical medical need and who may benefit from treatment prior to potential FDA approval in the United States. Vertex expects to open the expanded access program at clinical trial sites in the United States as early as July, pending FDA review and approval.
Vertex is working with regulatory authorities outside of the United States toward implementing additional expanded access programs in other countries, with a goal of opening programs for eligible patients in the second half of 2011.
For more information, please call Vertex Medical Information at 1-877-634-VRTX (8789).
- VX-770 Shows Positive and Lasting Results Throughout 48-Week Phase 3 Study
- Phase 2 Study of VX-770 and VX-809 Show Promising Results in Patients with Most Common Mutation
Source: Cystic Fibrosis Foundation and Vertex Pharmaceuticals