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FDA Approves Kalydeco (VX-770) — First Drug That Targets the Underlying Cause of Cystic Fibrosis

January 31, 2012

The Cystic Fibrosis Foundation today applauds 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 from the Cystic Fibrosis Foundation.

The FDA approved Kalydeco (kuh-LYE-deh-koh) for a segment of the CF population, those ages 6 and older with the G551D mutation of cystic fibrosis. The drug is taken in pill form.

“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 CF, and the science behind the drug has opened exciting new doors to research and development that may eventually lead to additional therapies that will benefit more people living with CF.”

The G551D mutation 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 CF Foundation played a significant role in the research and development of this groundbreaking new drug. “The unique and mutually beneficial partnership that led to the approval of Kalydeco serves as a great model for what companies and patient groups can achieve if they collaborate on drug development,” said FDA Commissioner Margaret A. Hamburg, M.D., in a news release issued by the FDA.

While Kalydeco represents a breakthrough for patients with the G551D mutation, it also represents hope for all people with CF, according to Preston W. Campbell, III, M.D., the CF Foundation’s executive vice president for medical affairs. “Our organization’s mission is to find a cure for cystic fibrosis, and we won’t stop our work until we reach our goal for all people living with the disease.”

The vast majority of individuals with CF in the United States – nearly 90 percent – have a different mutation, called Delta F508.

Developing therapies for patients with the Delta F508 mutation is among the current research priorities of the CF Foundation. 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.

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