Cystic Fibrosis Home > Kalydeco

If you have cystic fibrosis, your healthcare provider may prescribe Kalydeco. This medication comes as a tablet and is taken once daily, twice daily, or twice a week, depending on certain factors. Studies have shown that this medicine can help improve lung function, lessen symptoms of cystic fibrosis, and decrease pulmonary exacerbations. Side effects may include headaches, throat pain, and upper respiratory tract infections.

What Is Kalydeco?

Kalydeco™ (ivacaftor) is a prescription medication approved for the treatment of cystic fibrosis. Specifically, it is approved for use in adults and children as young as 6 years old who have certain mutations of the CF gene.
 
(Click Kalydeco Uses for more information on this topic, including possible off-label uses.)
 

Who Makes This Medication?

Kalydeco is manufactured for Vertex Pharmaceuticals Incorporated.
 

How Does Kalydeco Work?

In people with certain mutations of the CFTR (cystic fibrosis transmembrane conductance regulator) protein, the CFTR ion channels have a much lower likelihood of opening, compared to normal CFTR channels.
 
Kalydeco is a CFTR protein potentiator. This means that it helps to increase the probability that the CFTR channel in people with certain mutations opens to allow flow of chloride ions across cell membranes. This helps to correct the main problem, rather than just addressing symptoms, in people with this type of cystic fibrosis.
 

Clinical Effects

In studies, people who took Kalydeco had improved lung function, improved cystic fibrosis symptoms, and more weight gain, compared to people taking a placebo (a "sugar pill" that does not contain any active ingredients). Kalydeco also decreased the chance of pulmonary exacerbations.
 
Written by/reviewed by:
Last reviewed by: Kristi Monson, PharmD;
Last updated/reviewed:
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