Cystic Fibrosis Home > Kalydeco Uses

How Does It 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 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.

Is It Safe for Children to Use Kalydeco?

This drug is approved for use in children as young as six years old. This certainly does not mean that younger children cannot take Kalydeco; it just means that this medication has not been adequately studied in younger age groups. Talk with your child's healthcare provider about the particular benefits and risks of using this medication in children.

Off-Label Uses for Kalydeco

On occasion, healthcare providers may recommend medications for something other than the approved uses. This is called an "off-label" use. At this time, there are no well-accepted, off-label uses for Kalydeco.
Written by/reviewed by:
Last reviewed by: Kristi Monson, PharmD;
Last updated/reviewed:
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