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Orkambi

The ivacaftor/lumacaftor combination drug (OrkambiTM) is an oral medication for the treatment of cystic fibrosis. Orkambi targets the underlying cause of the disease in people with the most common CF gene mutation, F508del.

In 2015, the FDA approved the drug for people with CF ages 12 and older who have two copies of the F508del mutation.

Vertex Pharmaceuticals Inc. developed Orkambi with significant clinical, scientific and funding support from the Cystic Fibrosis Foundation. We played an important role in the research and development of both ivacaftor and lumacaftor, including initiating the efforts that led to the discovery of these small molecules.

For more information about CF drugs in development, visit the Foundation’s interactive pipeline.

  1. What is Orkambi and who can take the drug?
  2. How does Orkambi work?
  3. What CF mutations does Orkambi treat?
  4. Does Orkambi work in people with one copy of F508del?
  5. Is Orkambi being tested in people with other CF mutations besides F508del? 
  6. When will Orkambi be available for people with two copies of F508del younger than age 12?
  7. Will people with CF who take Orkambi still need other therapies? 
  8. Are there clinical trials underway for people with one copy of F508del?
  9. Is Orkambi a cure for cystic fibrosis? 
  10. How can I find out which CF mutations I or my child have?
  11. Will Orkambi be immediately available to those who need it? 
  12. If I am unable to afford Orkambi or do not have insurance, can I get assistance?
  13. What was the Foundation’s role in the research and development of Orkambi?


1. What is Orkambi and who can take the drug?

Orkambi is the brand name for the combination of ivacaftor (Kalydeco™) and lumacaftor. It is the first treatment that targets the underlying cause of CF in people with two copies of the F508del mutation, the most common CF mutation. Almost half of all people with cystic fibrosis in the United States have two copies of F508del. 

Orkambi is taken in pill form every 12 hours — once in the morning and once in the evening. The FDA has approved the drug for people with two copies of F508del ages 12 and older, representing nearly a third of individuals with CF in the United States, or about 8,500 people.

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2. How does Orkambi work?

Orkambi is taken in pill form every 12 hours — once in the morning and once in the evening. It is designed to help the defective CFTR protein function normally. 

The drug is a combination of ivacaftor (Kalydeco) and lumacaftor, two separate drugs taken as one pill. Both drugs are designed to address the underlying cause of CF — a defective protein, called CFTR, produced by mutations in the CF gene. 

In people with two copies of F508del, a series of problems prevents the CFTR protein from reaching the cell surface, where it is needed to help regulate the flow of salt and fluids in and out of the cells of the lungs and other organs.

Lumacaftor is designed to help move the defective CFTR protein to its proper place at the cell surface. Ivacaftor increases the activity of the protein once it is there, supporting the flow of salt and fluids.

In people with two copies of the F508del mutation, Orkambi improves lung function and other important health measures, such as weight gain.

Orkambi also significantly reduces the rate of pulmonary exacerbations, which often require hospitalization and IV antibiotics and can lead to a steep decline in lung function and cause permanent lung damage.

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3. What CF mutations does Orkambi treat?

Orkambi is designed for people with two copies of the most common CF mutation, F508del. The FDA has approved the drug for people with two copies of F508del ages 12 and older, representing nearly a third of those with CF in the United States.

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4. Does Orkambi work in people with one copy of F508del?

Orkambi was tested in people with CF with only one copy of the F508del mutation and was shown not to be effective. For more information about clinical trials for people with one copy of F508del, see question 8 below.

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5. Is Orkambi being tested in people with other CF mutations besides F508del?

Orkambi is currently not being tested in people with other CF mutations. It is possible that, based on further research, eligibility for Orkambi could eventually expand to other age groups or CF mutations, as we saw with Kalydeco. It is important to know that significant research is being done on other CF mutations in laboratories around the world. Our goal is to find treatments for all people with CF.

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6. When will Orkambi be available for people with two copies of F508del younger than age 12?

Vertex Pharmaceuticals Inc., the drug developer, is conducting a Phase 3 study of the therapy in children ages 6 to 11 who have two copies of the F508del mutation.

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7. Will people with CF who take Orkambi still need other therapies?

While Orkambi is an important new treatment for people with two copies of F508del, patients will still need daily treatments to help manage the symptoms of the disease. Before starting Orkambi, talk with your CF care team about how the drug fits into your treatment regimen.

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8. Are there clinical trials underway for people with one copy of F508del?

Vertex is conducting three Phase 3 trials of ivacaftor and another potential compound, VX-661, in people ages 12 and older who have one copy of the F508del mutation and a second CF mutation that is either a residual function mutationgating mutation or minimal function mutation.

If you have questions about whether one of these trials might be right for you or your child, speak with your CF doctor or call Vertex at 1-877-752-5933.

You can also learn more about these and other current CF clinical trials by visiting our drug development pipeline. Most CF trials underway now — including those studying new antibiotics, anti-inflammatory medications and nutritional strategies — are not limited to people with specific mutations of CF.

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9. Is Orkambi a cure for cystic fibrosis? 

No. While Orkambi improves lung function and other important health measures in people with two copies of the F508del mutation, it is not a cure. People with CF who take the combination drug will still need other daily treatments to manage the symptoms of the disease.

It is important to note that we can’t rely on any one treatment to win the battle against CF. That’s why the CF Foundation continues to invest in and explore all promising avenues of research to attack the disease from every angle.

The Foundation funds research programs to develop new therapies that target the underlying cause of CF as well as treatments to help people with CF manage the symptoms of their disease.

In addition, the Foundation has expanded its efforts to pursue promising new technologies that could address the disease at its most fundamental levels, including repair of the faulty CF gene itself. While these new approaches are in the earliest stages, they could one day lead to our ultimate goal of finding a one-time cure for all people with CF, regardless of an individual’s genetic mutations.

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10. How can I find out which CF mutations I or my child have?

If you do not know your or your child's CF mutations, contact your CF doctor or care center.

The CF Foundation’s Mutation Analysis Program (MAP) offers free and confidential genetic testing to patients with a confirmed diagnosis of cystic fibrosis. MAP provides genotyping for people with CF who have not yet been tested, or who have been tested previously but still have one or more unknown mutations.

To learn more about MAP, visit the Mutation Analysis Program page or talk with your CF doctor or care center.

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11. Will Orkambi be immediately available to those who need it?

For more information on obtaining Orkambi, please contact Vertex at 1-877-752-5933.

It is our hope that everyone prescribed this drug will have immediate access to it so they can begin to live longer, healthier lives. There are a number of factors that determine how soon a new drug becomes available after it’s approved. For many people, their health insurer plays a key role in when they can get a drug.

To address the issue of timely access to vital CF treatments, the Foundation educates health insurers about cystic fibrosis, and the role that specialty CF medications have played in adding decades of life for people with the disease. The Foundation convened a group of major payers in early 2015 for a forum focused on how we can collectively help people with CF get the treatments they need, when they need them.

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12. If I am unable to afford Orkambi or do not have insurance, can I get assistance?

For more information on obtaining Orkambi, contact Vertex by calling 1-877-752-5933 or visiting www.VertexGPS.com.

The Foundation’s Patient Assistance Resource Center (PARC) works with individuals and families to find the financial support and coverage they need to afford CF treatments. To contact PARC, call 1-888-315-4154 or email parc@cff.org.

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13. What was the CF Foundation’s role in the research and development of Orkambi?

The CF Foundation invests in the research and development of a wide variety of CF treatments, all in search of a cure and to help people with CF have the best health and quality of life. In the case of Orkambi, we provided significant scientific, clinical and financial support for discovery and development of ivacaftor and lumacaftor, including initiating the first studies that led to the discovery of these small molecules.

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