What is Orkambi (lumacaftor/ivacaftor) for?
Orkambi (lumacaftor/ivacaftor), a combination of two active ingredients, lumacaftor and ivacaftor, is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, indicated for the treatment of cystic fibrosis (CF) in patients who are homozygous for the F508del mutation in the CFTR gene1,2,3. This is the first medicine for cystic fibrosis directed at treating the cause of the disease in people who have this specific mutation4,5.
The dosage 200 mg/125 mg it is indicated for patients who are 12 years or older1,2,3. The dosage of 100 mg/125 mg, is indicated for patients aged 6 to 11 years1,2.
How does Orkambi (lumacaftor/ivacaftor) work?
Cystic Fibrosis (CF) is a serious genetic disorder characterised by the formation of thick mucus that accumulates in the lungs, digestive tract and other parts of the body leading to severe respiratory and digestive problems, as well as other complications such as infections and diabetes4. Mutations in the CFTR gene are responsible for this disease. This gene makes a protein called ‘cystic-fibrosis transmembrane conductance regulator’ (CFTR), which is involved in regulating the production of mucus and digestive juices. These mutations reduce the number of CFTR protein channels on the cell surface or affect the way they work. These channels are used for the transport of ions (charged atoms and molecules) in and out of cells. When the channels are defective, mucus and digestive juices can become abnormally thick and sticky5. Orkambi addresses this problem from two sides. One of the active substance, lumacaftor, increases the number of CFTR proteins on the cell surface and the other, ivacaftor, increases the activity of the defective CFTR protein. This normalises the transport of ions through channels making the secretions less thick5.
Where has Orkambi (lumacaftor/ivacaftor) been approved?
Orkambi (lumacaftor/ivacaftor) was approved for the treatment of patients with cystic fibrosis (CF) in presence of F508del mutation in the CFTR gene by:
Food and Drug Administration (FDA), USA, July 2, 20154
European Medical Agency (EMA), European Union, November 19, 20155
Therapeutic Goods Administration (TGA), Australia, March 3, 20163
Please note that this medicine may have also been approved in other regions than the ones we’ve listed. If you have a question about its approval in a specific country feel free to contact our support team.
How is Orkambi (lumacaftor / ivacaftor) taken?
The standard dosage for patients aged 12 years or older is:
- two tablets (each containing 200 mg of lumacaftor and 125 mg of ivacaftor) taken orally every 12 hours1,2,3
The standard dosage for pediatric patients aged 6 to 11 years:
- two tablets (each containing 100 mg of lumacaftor and 125 mg of ivacaftor) taken orally every 12 hours1,2.
Reduce dose in patients with moderate or severe hepatic impairment. When initiating Orkambi (lumacaftor / ivacaftor) in patients taking strong CYP3A inhibitors, reduce Orkambi (lumacaftor / ivacaftor) dose for the first week of treatment1.
Complete information about Orkambi (lumacaftor / ivacaftor) dosage and administration can be found in the official prescribing information listed in our resources section1,2,3.
Note: Please consult with your treating doctor for personalised dosing.
Are there any known side effects of Orkambi (lumacaftor/ivacaftor)?
The most common side effects were dyspnea, nasopharyngitis, nausea, diarrhoea, upper respiratory tract infection, fatigue, respiration abnormal, blood creatine phosphokinase increased, rash, flatulence, rhinorrhea, influenza1.
For a comprehensive list of side effects and adverse reactions please refer to the official prescribing information1,2,3.