Lumacaftor/ivacaftor

Chemical structures of Ivacaftor and Lumacaftor

Lumacaftor/ivacaftor is a combination medication used in the treatment of cystic fibrosis (CF) in patients who have specific genetic mutations. This medication is marketed under the brand name Orkambi and is produced by Vertex Pharmaceuticals.

Mechanism of Action

Lumacaftor/ivacaftor works by targeting the defective CFTR (cystic fibrosis transmembrane conductance regulator) protein, which is responsible for the symptoms of cystic fibrosis.

• Lumacaftor acts as a chaperone that helps the CFTR protein fold correctly and reach the cell surface. It is classified as a CFTR corrector.
• Ivacaftor is a CFTR potentiator that enhances the function of the CFTR protein once it is at the cell surface, improving chloride ion transport.

Together, these drugs improve the function of the CFTR protein in patients with the F508del mutation, the most common mutation causing cystic fibrosis.

Indications

Lumacaftor/ivacaftor is indicated for the treatment of cystic fibrosis in patients aged 2 years and older who are homozygous for the F508del mutation in the CFTR gene. This mutation is present in approximately 50% of cystic fibrosis patients.

Administration

The medication is administered orally in the form of tablets. The dosage is typically based on the patient's age and weight, and it is recommended to be taken with fat-containing food to enhance absorption.

Side Effects

Common side effects of lumacaftor/ivacaftor include:

• Respiratory tract infection
• Nasal congestion
• Diarrhea
• Nausea
• Rash

Serious side effects can include liver enzyme elevations and respiratory events, particularly in patients with advanced lung disease.

Related Pages

• Cystic fibrosis
• CFTR
• Vertex Pharmaceuticals
• Ivacaftor