Drugs Home > What Is Ivacaftor Used For?

Available by prescription, ivacaftor is used for the treatment of cystic fibrosis in adults and children as young as six years old. However, not everyone who has cystic fibrosis can benefit from this medication. Ivacaftor is only approved to treat this disease in people with a certain gene mutation.

An Overview of Uses for Ivacaftor

Ivacaftor (Kalydeco™) is a prescription cystic fibrosis (CF) medication. While it is an exciting breakthrough for CF treatment, ivacaftor is only effective for CF in people who have certain mutations, including G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R. Ivacaftor is not effective in people with the most common CF mutation (known as F508del mutation).

What Is Cystic Fibrosis?

Cystic fibrosis (CF) is an inherited disease of the mucus and sweat glands. While most people think of cystic fibrosis as a lung disease, it also affects the pancreas, liver, intestines, sinuses, and sex organs.
Normally, mucus is watery. It keeps the lining of certain organs moist, and prevents them from drying out or getting infected. However, in cystic fibrosis, an abnormal gene causes mucus to become thick and sticky (see Cystic Fibrosis Gene and Causes of Cystic Fibrosis). The thick, sticky mucus builds up in the lungs and blocks the airways. This makes it easy for bacteria to grow and causes the following cystic fibrosis symptoms:
  • Frequent coughing that brings up thick sputum, or phlegm
  • Frequent bouts of bronchitis and pneumonia, which can lead to inflammation and permanent lung damage.
Treatment for cystic fibrosis often includes several of the following options:
  • Medications, including antibiotics to treat lung infections
  • Chest physical therapy
  • Exercise
  • Oxygen
  • Lung transplantation.
It is also important for people with cystic fibrosis to take good care of themselves in general (see Living With Cystic Fibrosis for more information).
Ivacaftor is approved for cystic fibrosis treatment in people with a certain mutation. Tests might be necessary in order to check which type(s) of mutation(s) a person has before treatment with ivacaftor may begin. Also, ivacaftor is an exceptionally expensive medication. People deciding to begin treatment with this drug should check with their insurance, Medicaid, or Medicare, to see if it is covered.
Written by/reviewed by:
Last reviewed by: Kristi Monson, PharmD;
Last updated/reviewed:
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