The Cystic Fibrosis Foundation Applauds FDA Approval of Kalydeco™, First Drug to Address the Underlying Cause of Cystic Fibrosis

Kalydeco Marks the Latest Success of the Cystic Fibrosis Foundation’s Venture Philanthropy Model

Jan. 31, 2012 | 5 min read

(Bethesda, Md.) -- The Cystic Fibrosis Foundation today applauded the Food and Drug Administration's approval of Kalydeco™ (ivacaftor; previously known as VX-770), a major advance in the search for a cure for cystic fibrosis. The drug was developed by Vertex Pharmaceuticals Inc. with scientific, clinical and significant funding support -- $75 million -- from the CF Foundation.

Kalydeco (kuh-LYE-deh-koh) is a groundbreaking new drug in pill form that is the first to address the underlying cause of cystic fibrosis (CF). The FDA approved the drug for a segment of the CF population, those ages 6 and older with the G551D mutation of cystic fibrosis.

“Today marks an important milestone in our journey to find a cure for cystic fibrosis,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “Kalydeco addresses the underlying cause of cystic fibrosis, and the science behind the drug has opened exciting new doors that may eventually lead to a cure for all people living with this disease.”

“The Cystic Fibrosis Foundation is proud to have played a vital role in the research and development of this groundbreaking new drug, and we applaud the FDA for its swift approval,” Beall added.

Cystic fibrosis is a fatal genetic disease that causes serious lung infections and premature death. It affects about 30,000 children and adults in the United States and 70,000 people worldwide.

Kalydeco is the latest example of the CF Foundation's innovative venture philanthropy drug development model, through which it has invested hundreds of millions of dollars to help develop CF drugs and therapies. The model provides upfront funding for pharmaceutical companies to help ease the financial burden of developing drugs to treat rare diseases like this one. This innovative strategy has led to tremendous advances in life expectancy and quality of life for CF patients, and has helped to create nearly every drug and therapy available to treat cystic fibrosis.

The G551D mutation that Kalydeco targets is present in roughly 4 percent of the CF patient population in the United States. In people with this mutation, a defective protein caused by CF moves to its proper place at the surface of the cell but does not function correctly. Instead, the defective protein acts like a locked gate, preventing the proper flow of salt and fluid in and out of the cell.

Kalydeco helps unlock that gate and restore the function of the defective protein. The drug dramatically improves lung function, lowers sweat chloride levels and helps patients gain weight - all key clinical indicators of CF.

The vast majority of individuals with CF -- nearly 90 percent -- have a different mutation, called Delta F508.

“While the approval of Kalydeco represents a breakthrough for those patients with the G551D mutation, it also represents hope for all CF patients,” said Preston W. Campbell, III, M.D., executive vice president for medical affairs of the Cystic Fibrosis Foundation. “The CF Foundation is committed to finding a cure for CF, and the approval of Kalydeco marks an important first step in making that mission a reality.”

Current research priorities of the CF Foundation include developing therapies for individuals with the Delta F508 mutation, the most common CF mutation. Progress toward this goal includes an ongoing Phase 2 clinical trial of Kalydeco in combination with another potential therapy, VX-809, in people with the Delta F508 mutation. Results from the first part of this Phase 2 trial were positive. The second part of the study is now under way.

In addition, the CF Foundation has significantly expanded its research investments with other leading pharmaceutical companies, including Genzyme and Pfizer, to accelerate the discovery and development of new drugs that will help more CF patients. 

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The CF Foundation funds more CF research than any other organization, and nearly every CF drug available today was made possible because of CF Foundation support. Based in Bethesda, Md., the Foundation also supports and accredits a national care center network that has been recognized by the National Institutes of Health as a model of care for a chronic disease. The CF Foundation is a donor-supported nonprofit organization.  

Media Contact

Laurie Fink, national director of media relations:

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