Kalydeco (Ivacaftor (Ivacaftor) — Uses, Dosage, Side Effects & Safety

Drug class: Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Potentiator

Overview

Kalydeco (Ivacaftor) is a prescription medication used to treat certain types of cystic fibrosis (CF) in patients who have specific mutations in the CFTR gene. It is not suitable for all CF patients, but is effective for those with gating (Class III) mutations and some residual function mutations. Ivacaftor works by improving the function of the defective CFTR protein, helping to regulate the flow of salt and water in and out of cells. This can help reduce symptoms such as thick mucus in the lungs and improve lung function. Key points: Kalydeco is taken orally and is usually well tolerated. It is important to take it with a fatty meal for best absorption. Patients should have regular follow-up and monitoring while on this medication.

Therapeutic uses

  • Cystic fibrosis in patients aged 4 months and older who have at least one G551D mutation in the CFTR gene
  • Cystic fibrosis in patients aged 4 months and older who have at least one of 9 other specified gating (Class III) mutations in the CFTR gene
  • Cystic fibrosis in patients aged 4 months and older who have an R117H mutation in the CFTR gene
  • Cystic fibrosis in patients aged 4 months and older who have certain residual function mutations in the CFTR gene

Common side effects

  • Headache
  • Diarrhea
  • Nausea
  • Upper respiratory tract infection
  • Rash
  • Abdominal pain
  • Dizziness
  • Elevated liver enzymes

Frequently Asked Questions about Kalydeco (Ivacaftor

What is Kalydeco (Ivacaftor) used for?

Kalydeco is used to treat cystic fibrosis in patients with specific mutations in the CFTR gene. It helps improve lung function and reduce symptoms by enhancing the activity of the defective protein.

How should I take Kalydeco?

Take Kalydeco exactly as prescribed, with a fat-containing meal or snack, to ensure proper absorption. Do not skip doses and do not take extra doses to make up for missed ones.

What are the most common side effects?

Common side effects include headache, upper respiratory tract infection, stomach pain, diarrhea, and rash.

Can children take Kalydeco?

Yes, Kalydeco is approved for use in children as young as 6 months old, with dosing adjusted based on age and weight.

Do I need genetic testing before starting Kalydeco?

Yes, genetic testing is required to confirm that you have a CFTR mutation responsive to ivacaftor.

Are there any foods or drugs I should avoid?

Avoid grapefruit and Seville oranges, as well as strong CYP3A inducers like rifampin and carbamazepine, which can affect how Kalydeco works.

What should I do if I experience yellowing of the skin or eyes?

Contact your healthcare provider immediately, as this may be a sign of liver problems, a known risk with Kalydeco.

Educational information only. Always consult a qualified healthcare professional before starting, stopping, or changing any medication.