Lumacaftor/Ivacaftor (Orkambi (lumacaftor and ivacaftor) — Uses, Dosage, Side Effects & Safety

Drug class: Respiratory System

Overview

Lumacaftor/Ivacaftor (Orkambi) is a prescription medication used to treat cystic fibrosis (CF) in patients aged 2 years and older who have two copies of the F508del mutation in the CFTR gene. Cystic fibrosis is a genetic disorder that affects the lungs and digestive system, causing thick, sticky mucus to build up and leading to breathing and digestive problems. This medication combines two active ingredients: lumacaftor, which helps correct the folding of the defective CFTR protein, and ivacaftor, which improves the function of the protein at the cell surface. Together, they help improve lung function and reduce the frequency of pulmonary exacerbations. Key points for patients: Orkambi does not cure cystic fibrosis but can help manage symptoms and improve quality of life. It is important to take this medication exactly as prescribed and to continue other CF treatments as directed by your healthcare provider.

Therapeutic uses

  • Cystic fibrosis in patients aged 2 years and older who are homozygous for the F508del mutation in the CFTR gene

For related Lumacaftor/Ivacaftor (Orkambi guidance, compare Fluticasone + Salmeterol (Advair and Oxtriphylline.

Common side effects

  • Dyspnea
  • Nausea
  • Diarrhea
  • Upper respiratory tract infection
  • Rash
  • Chest discomfort
  • Liver enzyme elevations
  • Cataracts

Frequently Asked Questions about Lumacaftor/Ivacaftor (Orkambi

What is Lumacaftor/Ivacaftor (Orkambi) used for?

It is used to treat cystic fibrosis in patients aged 2 years and older who have two copies of the F508del mutation in the CFTR gene.

How does Orkambi work?

Orkambi combines two medicines: lumacaftor helps correct the misfolded CFTR protein, and ivacaftor improves its function at the cell surface, helping to reduce symptoms of cystic fibrosis.

What are the common side effects of Orkambi?

Common side effects include headache, nausea, diarrhea, upper respiratory tract infection, and increased sputum.

Can Orkambi be taken with other cystic fibrosis medications?

Yes, Orkambi is often used alongside other CF treatments, but always consult your healthcare provider for guidance.

What should I do if I miss a dose?

Take the missed dose within 6 hours if possible. If more than 6 hours have passed, skip the dose and continue your regular schedule. Do not double up.

Are there any foods or drugs to avoid while taking Orkambi?

Avoid grapefruit, Seville oranges, and strong CYP3A inducers like rifampin, as these can affect drug levels.

Is Orkambi safe during pregnancy or breastfeeding?

There is limited data on safety in pregnancy and breastfeeding. Discuss potential risks and benefits with your healthcare provider.

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