Vertex Pharmaceuticals Inc, Cambridge, Mass, recently announced plans to begin clinical trials of the potential cystic fibrosis (CF) therapy [removed]Kalydeco™ (VX-770)[/removed] in more patients groups— including children as young as 2 years old—later this year,.
Kalydeco is currently under review by the US Food and Drug Administration (FDA) for approval in people ages 6 and older with the G551D mutation of CF.
The FDA has granted Vertex a 6-month priority review of the drug, setting a target date of April 18, 2012, for the approval decision. If approved, Kalydeco would be the first drug available that targets the underlying cause of CF.
Vertex is planning clinical trials of Kalydeco in patient groups that were not evaluated in earlier Phase 3 studies of the drug, including children ages 2 to 5 with gating mutations, including G551D; people with other gating mutations besides G551D; and people with at least one copy of the R117H mutation of CF.
G551D is the most common gating mutation, accounting for 4% of people with CF in the United States. The remaining gating mutations are found in about 1% of the CF population. About 3% of people with CF in the United States have the R117H mutation.
Kalydeco is also being tested in an ongoing Phase 2 trial in combination with another potential therapy, VX-809, in people with the most common mutation of CF, Delta F508. In addition, Vertex plans to launch a clinical trial in mid-2012 studying Kalydeco with another therapy in development, VX-661, in people with the Delta F508 mutation.
The CF Foundation worked with Vertex to discover Kalydeco, VX-809, and VX-661 and provided substantial financial and clinical support throughout the development process.
Source: Cystic Fibrosis Foundation