William Elder, Jr., a medical student with cystic fibrosis, spoke at the event about the health gains he has experienced since starting Kalydeco, the first oral drug that treats the underlying cause of CF in a small group of people with the disease.
“Thanks to this treatment, I am able to attend medical school, and I'm healthier now than ever before in my life,” Elder said.
He also stressed the importance of keeping the
Called “Breakthrough Therapy Designation,” the new FDA program is designed to improve communication between the drug industry and the FDA, resulting in greater efficiency in the drug approval process. Kalydeco, in combination with the potential new drug VX-809, was the first therapy to receive the special designation. The combination therapy is currently in phase 3 clinical trials, and has the potential to help thousands with CF live healthier lives.
“The FDA's new breakthrough designation, combined with the work of the CF Foundation, creates a more defined and efficient development pathway for CF therapies,” said Robert J. Beall, Ph.D., president and chief executive officer of the Cystic Fibrosis Foundation. “This has attracted new investment and approaches to developing innovative drugs for CF and provides a roadmap for many other rare diseases.”
Elder was joined by Sen. Michael Bennet (D-CO), who provided the keynote address at the briefing, Jeffrey Leiden, the chief executive officer of Vertex Pharmaceuticals, Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research and other representatives from the medical community.
Watch a video of the briefing here.