Orkambi (lumacaftor/ivacaftor)

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Orkambi (lumacaftor/ivacaftor)

Orkambi (lumacaftor/ivacaftor) is a medicine indicated for the treatment of cystic fibrosis (CF) in patients who are homozygous for the F508del mutation in the CFTR gene.

Pulmonology Cystic Fibrosis
Marketing Authorisation Holder Vertex Pharmaceuticals (Europe) Ltd
Mode of Action CFTR potentiator (targeted therapy)
Administration Oral
Regulatory agency approval European Medical Agency (EMA), European Union, Food and Drug Administration (FDA), USA, Therapeutic Goods Administration (TGA), Australia
Strength 200mg/125mg milligram
Package 112 Tablets

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 FDA only approved dosage of 100 mg/125 mg, is indicated for patients aged 6 to 11 years1.

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 (FDA approved only):

  • two tablets (each containing 100 mg of lumacaftor and 125 mg of ivacaftor) taken orally every 12 hours1.

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.

More Information
Working ingredient lumacaftor/ivacaftor
Shipping method Controlled room temperature (temperature controlled)
Package 112
Therapeutic Area Respiratory
Pulmonology Cystic Fibrosis
Indication Cystic fibrosis with F508del mutation
Administration Oral
Mode of Action CFTR potentiator (targeted therapy)
Regulatory agency approval European Medical Agency (EMA), European Union, Food and Drug Administration (FDA), USA, Therapeutic Goods Administration (TGA), Australia
Marketing Authorisation Holder Vertex Pharmaceuticals (Europe) Ltd
HS Code N/A
CAS Number N/A

The approvals were based on two double-blind, placebo-controlled clinical trials involving a total of 1,108 participants with CF who were 12 years and older with the F508del mutation. In both studies, participants with CF who took Orkambi (lumacaftor/ivacaftor), two pills every 12 hours, demonstrated improved lung function compared to those who took placebo5. The most relevant measure for the evaluation of the study results after 24 weeks was the the percentage of predicted forced expiratory volume in one second (ppFEV), which is the amount of air a person can blow out in one second divided by the expected amount, calculated by taking into account several factors such as the person's sex and height6. After 24 weeks of treatment, patients who took Orkambi (lumacaftor/ivacaftor) had an average improvement in ppFEV of 2.41% more than those who took placebo in the first study, and 2.65% in the second study. Treatment with Orkambi (lumacaftor/ivacaftor) also led to a reduction of 39% in the number of exacerbations requiring hospital admission or antibiotic therapy5.

1. Summary of Product Characteristics [FDA]: Orkambi (lumacaftor / ivacaftor) [PDF]
    Vertex Pharmaceuticals Inc., Jan. 2018

2. Summary of Product Characteristics [EMA]: Orkambi (lumacaftor / ivacaftor) [PDF]
Vertex Pharmaceuticals Europe Ltd., Mar. 2018

3. Summary of Product Characteristics [TGA]: Orkambi (lumacaftor / ivacaftor) [PDF] 
Vertex Pharmaceuticals (Australia) Pty Ltd, Mar. 2016

4. FDA News Release: FDA approves new treatment for cystic fibrosis,
FDA, 02/07/2015

5. EMA. Human Medicines: Orkambi (lumacaftor/ivacaftor)
1/12/2015, (last update: 24/01/2017), cited on 20/06/2017

6. Naylor, M.G., et al. Recommendations for using standardised phenotypes in genetic association studies
Hum Genomics. 2009; 3 (4): 308–319

Shipping Type Cold Chain
Shipping method Controlled room temperature (temperature controlled)
Dynamic Weight No
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Disclaimer: Every effort has been made to ensure that the information provided here is accurate, up-to-date and complete, but no guarantee is made to that effect. Medicine information contained herein may be time sensitive. The absence of a warning for a given medicine or combination thereof in no way should be construed to indicate that the medicine or combination is safe, effective or appropriate for any given patient. Always consult your treating physician before starting a course of treatment.