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FDA Approves Expanded Use of Kalydeco™ for Eight Additional Mutations that Cause CF

February 21, 2014

The U.S. Food and Drug Administration announced today it has approved Kalydeco™ to treat people ages 6 and older who have one of eight additional cystic fibrosis mutations.

With the announcement today, Kalydeco is now approved for people with the following mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D. In 2012, the FDA approved Kalydeco for use in people with the G551D mutation ages 6 and older.

In the United States, approximately 150 people have one of the additional eight mutations for which Kalydeco is now approved. Kalydeco, a breakthrough therapy, is the first drug to treat the underlying cause of cystic fibrosis.

“Kalydeco’s expanded approval is welcome news for the CF community,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “We continue to support research with Kalydeco in more patient groups, and at the same time, are aggressively pursuing the development of other promising compounds that treat the root cause of CF and benefit the greatest number of people with cystic fibrosis.”

In the group of eight mutations, and G551D, the defective protein in CF moves to its proper place at the surface of the cell but does not function correctly. Instead, it acts like a locked gate. Kalydeco is designed to unlock that gate, allowing the proper flow of salt and water in and out of the cells lining the airways and other organs.

The FDA expanded Kalydeco’s use based on results of a Phase 3 clinical trial of the drug in people with these mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or G970R.  Study participants who took Kalydeco showed statistically significant improvements in lung function, Body Mass Index and other measures, compared with those who received the placebo.

Data from the study did not support approval of the drug in patients with the G970R mutation. Vertex estimates that about 10 people worldwide have this mutation, including two in this country.

Kalydeco was developed by Vertex Pharmaceuticals, with a $75 million investment from the CF Foundation, which was used to discover this drug and other potential treatments still in development. 

The Foundation is investing in a robust pipeline of potential therapies that target the disease from every angle, including research collaborations with Pfizer, Genzyme and Vertex to accelerate the discovery and development of new drugs.

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