What is Orkambi? What is Orkambi used for?

Orkambi is a combination of two drugs, lumacaftor and ivacaftor (Kalydeco) and is used for treating cystic fibrosis. Cystic fibrosis (CF) is an inherited disease that affects the secretory glands, including the mucus and sweat glands. Cystic fibrosis mostly affects the lungs, pancreas, liver, intestines, sinuses, and sex organs. Cystic fibrosis is due to a mutation in the cystic fibrosis gene on chromosome 7. The cystic fibrosis gene encodes a protein known as the cystic fibrosis transmembrane regulator (CFTR). The abnormal CFTR protein in patients with cystic fibrosis leads to disruption of chloride channels on the cells which are important for the secretion of water. Cystic fibrosis is characterized by the production of abnormal mucus that is excessively thick and sticky because it lacks water. The abnormal mucus leads to blockages within the airways of the lungs. This leads to repeated, serious lung infections that can permanently and progressively damage the lungs.

Lumacaftor corrects abnormalities in the CFTR protein and increases the amount of functional CFTR protein in one type of mutation. Ivacaftor increases the action of CFTR protein. The combination of both drugs increases the quantity, stability, and function of CFTR, reducing the production of abnormal mucous and increasing lung function. The FDA approved Orkambi in July, 2015.

What brand names are available for Orkambi?


Is Orkambi available as a generic drug?


Do I need a prescription for Orkambi?


What are the side effects of Orkambi?


  • Orkambi should be used with caution in patients with advanced liver disease and only when the benefits are expected to outweigh the risks.
  • Orkambi is associated with liver-related adverse events. Liver function should be tested before treatment and every 3 months during the first year of treatment, and yearly thereafter.
  • Chest discomfort, dyspnea, and abnormal respiration may occur.
  • Non-congenital lens opacities/cataracts have been reported in pediatric patients treated with ivacaftor. Baseline and follow-up eye examinations are recommended in pediatric patients treated with Orkambi.

What is the dosage for Orkambi?

Adults and pediatric patients age 12 years and older should take two tablets every 12 hours with fat-containing food since fat increases the absorption of Orkambi. Examples of fat-containing foods include

  • eggs,
  • avocados,
  • nuts,
  • butter,
  • peanut butter,
  • cheese pizza, and
  • whole-milk dairy products such as whole milk, cheese, and yogurt.

If a dose is missed it may be taken within 6 hours of the regular dosing time. If more than 6 hours has passed since the regular dosing time, the dose should be skipped and taken at the next regular scheduled time. The dose should not be doubled.

Which drugs or supplements interact with Orkambi?

: Orkambi may decrease blood levels and reduce the effect of some drugs that are broken down by the liver. Examples of interacting drugs include

Blood levels of digoxin and effect of warfarin should be monitored closely because Orkambi may affect blood levels of digoxin and warfarin.

Is Orkambi safe to take if I'm pregnant or breastfeeding?

Orkambi is probably excreted in human milk. The effect of Orkambi on nursing infants has not been evaluated.

What else should I know about Orkambi?

What preparations of Orkambi are available?

Tablets: lumacaftor 200 mg and ivacaftor 125 mg

How should I keep Orkambi stored?

Orkambi should be stored at room temperature, between 15 C and 30 C (59 F and 86 F).


Lumacaftor and ivacaftor (Orkambi) is a combination drug prescribe to treat cystic fibrosis (CF) in patients 12 years of age and older who are homozygous. Side effects, multiple drug interactions, dosage, and pregnancy and breastfeeding safety should be reviewed prior to taking this medication.

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