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What Is Kalydeco (Ivacaftor)?

Cystic fibrosis (CF) is a genetically inherited disease that results in persistent lung infections and gastrointestinal issues that gradually damage many organs. People with CF inherit two copies of a defective or mutated CF gene, meaning each parent was a carrier for CF.

CFTR gene and cystic fibrosis

Called the cystic fibrosis transmembrane conductance regulator (CFTR) gene, this mutation affects production of the CFTR protein. A properly made CFTR protein helps cells move chloride, an element in salt, to the cell surface. In CF, the CFTR protein malfunctions can’t move chloride properly. Without chloride, water isn’t attracted to the cell surface, resulting in a thick, sticky mucus building up in the lungs and other organs.

A class of drugs called CFTR modulators target specific defects in the CFTR protein so that it can work properly. One such drug is ivacaftor, which goes by the brand name Kalydeco.

How does Kalydeco work?

Ivacaftor is a potentiator and it helps people with CF who have what are known as gating and conductive mutations, and a few residual function and splice mutations. In these types of mutations, some CFTR protein reaches the surface of the cell. However, either not enough protein gets to the cell surface, or the protein does not let enough chloride through. Ivacaftor binds to the mutated CFTR protein at the cell surface and opens the gate, allowing chloride to flow through. This reduces the symptoms of CF.1

What types of cystic fibrosis can Kalydeco treat?

Ivacaftor used alone works for about 8 percent of those with CF. It can be used by people with CF who are 6 months or older who have one of the following CFTR mutations2:

Gating Mutations

G178R
G1244E
S549R
G551D
G1349D
S1251N
G551S
S549N
S1255P
Residual Function Mutations

A455E
E193K
R117C
A1067T
F1052V
R347H
D110E
F1074L
R352Q
D110H
G1069R
R1070Q
D579G
K1060T
R1070W
D1152H
L206W
S945L
D1270N
P67L
S977F
E56K
R74W
Splice Mutations

711+3A→G
3272-26A→G
E831X
2789+5G→A
3849+10kbC→T
Conductor Mutation

R117H

 

How is Kalydeco taken?

Ivacaftor is prescribed to people with CF who are age 1 and older. People with CF who are age 6 and older, take one ivacaftor tablet by mouth every 12 hours, with foods that contain fat such as eggs, avocado, peanut butter, whole milk, cheese or nuts.

Children ages 1 to 5 take a dose based on their weight. For young children, the drug comes in a packet that is mixed with soft food or a liquid that contains fat. Ivacaftor may be combined with lumacaftor or tezacaftor to help people with other types of CFTR protein mutations.1-3

What are the risks or common side effects with Kalydeco?

The most common side effects of ivacaftor (Kalydeco) are:

  • Headache
  • Upper respiratory tract infections such as colds, sore throat, nasal and sinus congestion or runny nose
  • Stomach pain
  • Rash
  • Nausea
  • Dizziness2,3

Why genetic testing is important in cystic fibrosis

CFTR modulators are designed to correct the specific type of malfunction that certain CF mutations cause. If you do not have one of these mutations, ivacaftor will not work to correct why your CFTR protein fails to carry chloride through cells. This is why it is important to know which genetic mutation of CF you have. If you do not know your mutation, the CF Foundation offers free genetic testing for people with cystic fibrosis.

Written by: Jessica Johns Pool | Last reviewed: September 2019
  1. Cystic Fibrosis Foundation. CFTR Modulator Types. Available at: https://www.cff.org/Research/Developing-New-Treatments/CFTR-Modulator-Types. Accessed 5/29/2019.
  2. Cystic Fibrosis Foundation. CFTR Modulator Therapies. Available at: https://www.cff.org/Life-With-CF/Treatments-and-Therapies/Medications/CFTR-Modulator-Therapies. Accessed 5/29/2019.
  3. U.S. National Library of Medicine Daily Med. Kalydeco-ivacaftor tablet, film coated Kalydeco-ivacaftor granule. Available at: https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=0ab0c9f8-3eee-4e0f-9f3f-c1e16aaffe25. Accessed 5/29/2019.