The Foundation has nearly 30 drugs in development or already on the market for treating CF, a rare,
At the event, National Institutes of Health Director Francis Collins, M.D., Ph.D., noted that 25 million Americans suffer from rare diseases. “How can we take this wonderful CF Foundation success and spread it around?” he asked.
To achieve this goal, Collins has proposed a new
The briefing highlighted a collaboration between the CF Foundation and Vertex Pharmaceuticals, Inc., which has spawned two potential oral drugs that treat the root cause of CF - a historic turning point. All therapies available to patients today can only treat the symptoms of the disease. One of these potential drugs, VX-770, completed Phase 3 clinical trials this spring, with very promising results. Vertex will submit a
Vertex Chairman and CEO Matthew W. Emmens and Vertex Vice President and Cystic Fibrosis Franchise Lead Eric Olson, Ph.D., spoke at the briefing, along with Reps. Edward Markey (D-MA), Brian Bilbray (R-CA), Tom Marino (R-PA) and Jim McGovern (D-MA).
“We've come a long way” in treating cystic fibrosis, said Markey, who is co-chair of the Congressional Cystic Fibrosis Caucus. He wrapped up the briefing by saying: “It was a perfect hour of optimism.”