CF Foundation Drug Development Model Showcased at Congressional Briefing

June 14, 2011

Congressional Briefing
Robert J. Beall, President and CEO of the CF Foundation, spoke at a congressional briefing on the need to accelerate development of new drugs for rare diseases, along with Vertex Chairman and CEO Matthew W. Emmens (left) and Rep. Edward Markey (center).

The Cystic Fibrosis Foundation’s successful business model was at the center of a congressional briefing in Washington, D.C., today, which focused on strategies for jump-starting drug development for rare diseases.

The Foundation has nearly 30 drugs in development or already on the market for treating CF, a rare, genetic disease. It has created these opportunities by offering financial incentives to biotech companies to entice them to pursue CF drug development, said Robert J. Beall, Ph.D., president and CEO of the CF Foundation.

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 NIH initiative called the National Center for Advancing Translational Sciences. It will focus on taking scientific discoveries and promoting the translation of this knowledge into drugs for people in need, using some of the same strategies created by the CF Foundation.

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 New Drug Application to the FDA for VX-770 later this year.

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.”