70,000 children and young adults suffer from cystic fibrosis, an invariably fatal genetic disease. Every day that passes while they have no treatment results in permanent damage to the lungs, liver, pancreas, and other organs.
The FDA should offer Accelerated Approval for Lumacaftor and VX-661 in cystic fibrosis children with one or two copies of the delta-F508 mutation so that damage to their organs can be minimized while confirmatory trials are ongoing. These drugs are known to be effective in reversing the genetic defect and have been safely used in multiple clinical trials.
Our children are permanently injured by every day of delay. They cannot wait.