How KALYDECO® (ivacaftor) Works
KALYDECO affects gating of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. KALYDECO is used in patients age 2 years and older who have one of the following mutations in their cystic fibrosis (CF) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H.*
In patients with a G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H mutation in their CF gene, KALYDECO helps the “gates” open more often, allowing more chloride ions to move into and out of the cells. The movement of chloride ions may help keep a balance of salt and water in the lungs.
*The R117H mutation causes both a gating and a conductance defect. KALYDECO only works to improve CFTR protein gating, so it has no effect on the R117H mutation conductance defect.
What is known about how KALYDECO works was learned from studies conducted in a lab. Keep in mind that results from laboratory studies do not always match how these medicines work in a person. If you have any questions about your treatment, please speak with your doctor.
Understanding KALYDECO: A Video Series
Watch to learn about KALYDECO, including who it’s for, how it works, and how it’s taken.
This video series is intended to help you understand more about CF and treatment with KALYDECO. However, it should not replace discussions with your healthcare providers. Talk with your doctor if you have any questions about KALYDECO or CF.
What is KALYDECO® (ivacaftor)?
KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 2 years and older who have one of the following mutations in their CF gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R.
KALYDECO is used for the treatment of CF in patients age 2 years and older who have an R117H mutation in their CF gene.
KALYDECO is not for use in people with CF due to other mutations in the CF gene. KALYDECO is not effective in patients with CF with two copies of the F508del mutation (F508del/F508del) in the CF gene.
It is not known if KALYDECO is safe and effective in children under 2 years of age.
Important Safety Information
Who should not take KALYDECO?
Do not take KALYDECO if you take certain medicines or herbal supplements such as: the antibiotics rifampin (Rifamate®, Rifater®) or rifabutin (Mycobutin®); seizure medications such as phenobarbital, carbamazepine (Tegretol®, Carbatrol®, Equetro®) or phenytoin (Dilantin®, Phenytek®); or St. John’s wort.
Talk to your doctor before taking KALYDECO if you take any of these medicines or supplements.