Today, the U.S. Food and Drug Administration (FDA) approved the use of ivacaftor (Kalydeco®) for children with cystic fibrosis ages 1 to 2 who have at least one mutation that is responsive to ivacaftor. This includes the 38 mutations already approved for children ages 2 and older.

Ivacaftor is a type of cystic fibrosis transmembrane conductance regulator (CFTR) modulator called a potentiator that increases activity of the CFTR protein at the cell surface and has been shown in studies to improve lung function.

“Today's approval means we can work to prevent the onset of lung disease in eligible children as early as their first birthday,” said Michael P. Boyle, M.D., senior vice president of therapeutic development for the Cystic Fibrosis Foundation. “In the next few years, we hope these life-changing treatments that target the underlying cause of the disease are expanded to more than 90 percent of people with CF.”

This news follows the FDA's approval on Aug. 7 of the use of lumacaftor/ivacaftor (Orkambi®) for children with CF ages 2 to 5 who have two copies of the most common CF gene mutation, F508del. That expansion allowed 1,300 additional children in the U.S. to become eligible to receive the drug.

The approval of ivacaftor was informed by a positive study in children ages 1 to 2. Ivacaftor was generally well tolerated in the 24-week study, and there were no new safety concerns. Although safety was the primary focus of the study, it also found that the children's sweat chloride was significantly reduced to within the normal range during the trial.

In addition, the children in the study had substantial improvements in their levels of fecal elastase (a type of digestive enzyme). Fecal elastase levels are often used as a measurement of how well the pancreas is working. These data suggest that early treatment with ivacaftor may help preserve pancreatic function. A study of ivacaftor in infants ages 1 and younger is ongoing.

Ivacaftor was developed by Vertex Pharmaceuticals Inc. with significant clinical, scientific, and financial support from the Cystic Fibrosis Foundation.

The Foundation also supports research aimed at discovering new treatments for people with nonsense and rare mutations that do not produce the CFTR protein, an estimated 5 to 7 percent of people with CF. These individuals will be unable to benefit solely from CFTR modulators. In 2016, the Foundation launched the Nonsense and Rare Mutations Research and Therapeutics Initiative to help advance research by academic institutions and pharmaceutical companies focused exclusively on the creation of therapies to “restore” CFTR production for these individuals.