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Phase 3 Combination Studies of Ivacaftor (Kalydeco™) and Lumacaftor (VX-809) Show Positive Results in Most Common CF Mutation

June 24, 2014

Phase 3 clinical trials of ivacaftor (Kalydeco™) in combination with lumacaftor (VX-809) for people with two copies of the F508del mutation of cystic fibrosis showed significant improvements in lung function and other key measures of the disease, according to Vertex Pharmaceuticals Inc.

Based on these results, Vertex plans to submit a New Drug Application (NDA) by the end of 2014 to the U.S. Food and Drug Administration (FDA) for review, with possible approval in 2015. 

Results from the two 24-week clinical trials mark an important milestone for nearly 50 percent of people with cystic fibrosis — specifically those with two copies of F508del, the most common CF mutation. The potential treatment is the first to combine two pills to address the underlying genetic cause of CF in people with the F508del mutation. 

“This is a proud day for the CF community,” said Robert J. Beall, Ph.D., president and CEO of the CF Foundation. “Many people with CF and their families have been eagerly awaiting these results, and we are thrilled with the outcome. These studies further validate that we are on the right track to getting new and effective treatment into the hands of people with CF who so desperately need them.”

The Phase 3 trials were conducted at approximately 200 clinical trial sites in North America, Europe and Australia. In total, more than 1,100 people with two copies of the F508del mutation, ages 12 and older, participated in the studies.

The trials tested two different doses of the ivacaftor and lumacaftor combination therapy. 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. 

Repairing the defective protein in people with the F508del mutation is a particularly challenging and intricate process. In this mutation, a series of problems prevents the protein from reaching the surface of the cell. Lumacaftor is designed to move the F508del CFTR protein to the cell surface where ivacaftor can improve its function and help increase the normal flow of salt and fluids in and out of the cell.

These trials are the latest example of the CF Foundation’s innovative venture philanthropy model, in which the Foundation raises and invests millions of dollars in research toward new treatments. This model has led to tremendous advances in the health and quality of life for people with CF. In fact, the life expectancy of a child with CF has doubled in the last 30 years.

Ivacaftor and lumacaftor were discovered by Vertex in collaboration with the CF Foundation, which provided substantial clinical and financial support in the development process, including approximately $75 million in research funding. The Foundation has since committed another $75 million with Vertex for the discovery of additional CF drugs.

Ivacaftor was approved by the FDA as a stand-alone drug for people with the G551D mutation in 2012, and eligibility for the drug was expanded to people with several other closely related mutations in February 2014.

Dr. Beall continued: “Kalydeco's approval was the essential first step in treating the underlying defect in CF; now, with this promising drug combination, we are taking another critical step forward in making sure that all people with CF have effective treatments, regardless of their mutation. We will not rest until we reach that goal.”

The Foundation continues to pursue the development of other promising compounds that address the basic defect in CF through collaborative efforts with leading biotech and pharmaceutical companies, including Pfizer, Genzyme and others. The exciting gains in CF drug discovery and development have attracted considerable interest from pharmaceutical companies new to the CF field, spurring research and innovative strategies that could speed development of therapies for all people with CF, including those with rare mutations.

Additional Resources

  • Read the Vertex press release.

  • Learn more about the Phase 3 results in these FAQs.

  • Read a letter to the community from Foundation President and CEO Robert J. Beall, Ph.D., and Executive Vice President for Medical Affairs, Preston W. Campbell, III, M.D.

  • People with CF and their families who have questions about the Phase 3 results can contact Vertex Medical Information at 1-877-634-8789.
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