The studies showed that, compared with those on
These positive results represent an important milestone in the history of CF -- and a significant step forward in our effort to bring new treatments targeting the underlying cause of the disease to all people with CF.
Based on these results, Vertex plans to submit a
We congratulate an extraordinary community of people who contributed to the discovery and development of this combination treatment and helped bring us to this incredible day: the scientists who laid the foundation for our understanding of the
Equally important, we want to thank the tens of thousands of families, volunteers, donors, friends and staff who contributed their time, talent and dollars to help support the early discovery and development of this potential treatment.
The combination therapy ultimately aims to treat those with two copies of the F508del mutation or nearly 50 percent of the CF population. If it is approved, and with Kalydeco already available as a single therapy to a small segment of the CF population, the CF community could have effective drugs aimed at the root cause of CF for more than half of those living with the disease
Many of you have asked whether the drug combination might be made available before its potential FDA approval to people who are seriously ill, including those on lung transplant lists. We are pleased that Vertex is exploring options for an
These results further
We are encouraged that Vertex plans to conduct studies testing ivacaftor with another potential compound, called VX-661, in people with one copy of the F508del mutation.
We are also actively funding Vertex and other leading pharmaceutical and biotech companies - including Pfizer and Genzyme - to discover the next generation of CF therapies that can provide even more robust benefits to the health and
We hope in the meantime you will take a moment to reflect on today's exciting news and the important milestone it represents in our shared work to end cystic fibrosis. We know that with your dedication, passion and support, we will continue to find new ways to help advance new treatments for all people with the disease.
Robert J. Beall, Ph.D.
President and CEO
Preston W. Campbell, III, M.D.
Executive Vice President for Medical Affairs