FDA Approves Ivacaftor for Children Ages 2 to 5 with Certain Rare CF Mutations
March 18, 2015 | 3 min read

(Bethesda, Md.) -- The U.S. Food and Drug Administration (FDA) announced today that it has approved the use of ivacaftor (Kalydeco™) to treat cystic fibrosis in children ages 2 to 5 who have at least one of ten rare CF mutations.

For children who can't swallow a tablet, the drug will be available in the form of granules to be mixed with food or drink and available in two dosages based on the child's weight.

“We commend the FDA's decision to approve ivacaftor for a greater number of children living with cystic fibrosis,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “The Foundation is moving forward vigorously to pursue therapies that target the root cause of the disease for all people with CF.”

With this announcement, ivacaftor is now approved for people with CF ages 2 and older with at least one of the following mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D and R117H.

The total number of people with CF who are now eligible for the drug in the United States is 1,950 including 300 children ages 2 to 5. Ivacaftor was first approved in 2012 for people with the G551D mutation of CF ages 6 and older.

Developed by Vertex Pharmaceuticals Inc. with significant clinical and financial support from the CF Foundation, the drug is the first to target the underlying cause of the disease -- a defective CFTR protein caused by mutations in the CF gene.

In people with CF whose mutations can be treated by ivacaftor, the defective CFTR protein reaches the cell surface but does not function properly, interfering with the proper flow of salt and water in and out of the cell. Ivacaftor helps restore the function of the faulty CFTR protein.

Read Vertex's press release.

About the Cystic Fibrosis Foundation

The Cystic Fibrosis Foundation is the world's leader in the search for a cure for cystic fibrosis. The Foundation funds more cystic fibrosis research than any other organization, and nearly every CF drug available today was made possible because of 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 Cystic Fibrosis Foundation is a donor-supported nonprofit organization. 

Media Contact

Laurie Fink, National Director of Media Relations, Cystic Fibrosis Foundation: 301-841-2602lfink@cff.org

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