Cystic Fibrosis Drug Ivacaftor Approved for Young Children

A drug that has been designed to treat cystic fibrosis is in the news. Kalydeco is the brand name of the drug, and Ivacaftor is the generic name. The drug has been approved for patients who are 2 years old (or older) and who had specific mutations in their CF gene.

Kalydeco is a prescription medication that is used to treat cystic fibrosis (CF) in patients who are 2 years old (or older) and who have one of the following mutations in their CF gene: G551D, G1244E, Gi349D, G178R, G551S, S1251N, S1255P, S549N, or S549R. The official Kalydeco website also says the drug is used on CF patients who are 2 years old (or older) and who have an R177H mutation in their CF gene.

Cystic fibrosis (CF) is a genetically heritable disease that does not have a cure. CF causes severe damage to the lungs and digestive system. The disease affects cells that produce mucus, sweat and digestive juices. It makes those secretions thick and sticky. Those secretions are supposed to act as a lubricant in the body. The result is the thick secretions plug up tubes, ducts, and passageways in the body, especially in the lungs and pancreas.

Ivacaftor was approved in 2012 for treatment in children ages 6 and older, teens, and adults who had CF with a mutation on one of the genes listed above. In January of 2016, a study was done to see if ivacaftor was safe and effective on children who had CF and who were between the ages of 2 and 5.

The study included 34 cystic fibrosis patients who were between the ages of 2 and 5. Each child had at least one copy of a mutation in a gene linked to cystic fibrosis called the CFTR gene. The children took two daily doses of ivacaftor for six months. The amount they took was based on each child’s weight.

Overall, the drug was generally well tolerated by the children. Five children had liver function abnormalities and needed to stop treatment. (The damage was reversible.) It was believed that the study showed that ivacaftor was safe for kids who were at least 2 years of age. There was some question about exactly what the earliest age a child could be given ivacaftor.

Kalydeco is made by a company called Vertex Pharmaceuticals. The company applied to the FDA for approval of ivacaftor in CF patients over the age of 2 who had one of 23 residual function mutations in the CFTR gene. That application expanded the number of CF gene mutations that ivacaftor could be used to treat.

The FDA has denied the expanded use of the drug. That means ivacaftor has been approved for use on CF patients ages 2 and older who have one of the original gene mutations the drug currently treats.

Image by Hey Paul Studios on Flickr.

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