VX809 Breakthrough

Letter to the Community on Exciting Vertex Clinical Trials Results

June 24, 2014

This morning, Vertex Pharmaceuticals announced the results from the Phase 3 clinical trials of ivacaftor (Kalydeco™) and lumacaftor (VX-809) in people with two copies of the F508del mutation.

The studies showed that, compared with those on placebo, participants who took the combination treatment showed significant and consistent improvement in lung function and in other important health measures, including weight gain, and a reduction in the rate of pulmonary exacerbations.

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 New Drug Application to the U.S. Food and Drug Administration (FDA) by the end of this year. While we cannot predict the outcome of the FDA’s review, we believe the Phase 3 results present a compelling case for the approval of this much-needed potential new treatment.

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 genetic defect in CF, the researchers at Vertex and the clinical research teams who helped move these critical studies forward in record time and, especially, the more than 1,100 people with CF around the world who took part in the trials.  

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 expanded access (sometimes called compassionate use) program for those with two copies of F508del who could safely benefit from the combination therapy during the FDA review period.

These results further validate that we are on the right track to finding new treatments targeting the root cause of the disease. Even as we celebrate today’s announcement, we are determined to press ahead speedily in our pursuit of new therapies for all people with CF, including those with rare mutations, and we are making steady progress on a number of fronts.

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 quality of life for those with the disease. We look forward to sharing more about the progress of these and other research initiatives.

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.

Sincerely,

Robert J. Beall, Ph.D.		Preston W. Campbell, III, M.D.
President and CEO		Executive Vice President for Medical Affairs