Vertex Pharmaceuticals Inc. today announced final results of a Phase 3 clinical trial of VX-770, a CF medicine in development that targets the underlying cause of the disease. Participants who took the drug had rapid and significant improvements in lung function and other key measures of the disease, and they maintained these benefits throughout the 48-week study.
The trial evaluated VX-770 in people with CF age 12 and older who carry at least one copy of the G551D mutation. The results confirm preliminary findings released in February 2011.
On average, compared with patients on placebo, those receiving the drug had a marked increase in lung function, improved weight gain and a reduction in sweat chloride toward normal levels. Excessive sweat chloride is a key clinical indicator of CF.
The company also announced that results from a 48-week Phase 3 trial of VX-770 in children age 6 to 11 with the G551D mutation were consistent with positive 24-week results released earlier this year. In the children’s trial, those taking the drug showed similar improvements in lung function, weight gain and sweat chloride levels.
Vertex is on track to submit a New Drug Application for VX-770 to the U.S. Food and Drug Administration (FDA) in the second half of the year. Generally, the FDA takes between 6 and 12 months to review and rule on a drug application.
- Vertex to Expand Access to VX-770 for Patients with Critical Medical Need
- Phase 2 Study of VX-770 and VX-809 Show Promising Results in Patients with Most Common Mutation
Source: Cystic Fibrosis Foundation