The U.S. Food and Drug Administration (FDA) has approved the use of ivacaftor (Kalydeco®) for babies with cystic fibrosis ages 6 to 12 months who have certain mutations.
Ivacaftor is a CFTR modulator that increases activity of the CFTR protein at the cell surface and has been shown to improve lung function. The CFTR protein is shaped like a tunnel that can be closed by a gate. Ivacaftor holds the gate open so chloride can flow through.
Ivacaftor was initially approved in 2012 for treatment of people ages 6 and older with the gating mutation G551D, who made up 4 percent of the 30,000 people with CF in the U.S. Last year, approval was expanded to people ages 12 months and older with one of 38 mutations, who make up 16 percent of the CF population.
During a 24-week study in 11 children between 6 and 12 months, ivacaftor was generally well tolerated, and there were no new safety concerns, according to Vertex Pharmaceuticals. Although safety was the primary focus of the study, it showed that the children's sweat chloride was significantly reduced to within the near normal range during the trial.
“This expansion is great news for our community, as more people with CF will have this important treatment available earlier in life,” said Michael P. Boyle, M.D., senior vice president of therapeutics development for the Cystic Fibrosis Foundation. “The data from this study and others support our belief that starting on modulators at a young age has the potential to help slow or even prevent progression of the disease.”
Ivacaftor was developed by Vertex Pharmaceuticals Inc. with significant clinical, scientific, and financial support from the Cystic Fibrosis Foundation.
The Foundation is committed to ensuring that everyone with CF has a treatment for the underlying cause of their disease, including people with mutations who are unlikely to benefit from CFTR modulators (an estimated 7 percent of people with CF). Learn more about Research We Fund.
For more information, see the Vertex press release.