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Kalydeco™

Kalydeco™ is a new oral medication for the treatment of cystic fibrosis, approved by the U.S. Food and Drug Administration (FDA) in January 2012.

It is the first drug available that targets the underlying cause of CF — a faulty gene and its protein product, CFTR.

The FDA approved Kalydeco (kuh-LYE-deh-koh) for people ages 6 and older with the G551D mutation of CF.

The drug was formerly called VX-770 and is also known by its generic name, ivacaftor.

Vertex is working to study Kalydeco in certain people with CF that were not studied earlier, including children as young as age 2 with the G551D mutation.

Kalydeco has also been studied in a Phase 2 clinical trial in combination with another oral medication, VX-809, in people with the Delta F508 mutation of CF.

In addition, Vertex is enrolling people with two copies of the Delta F508 mutation for a Phase 2 study of Kalydeco with VX-661, a CF drug in development, in people with two copies of the Delta F508 mutation.

Vertex Pharmaceuticals Inc. developed Kalydeco, VX-809 and VX-661 with significant scientific, clinical and financial support from the Cystic Fibrosis Foundation.


What is Kalydeco™?

Kalydeco (kuh-LYE-deh-koh) is an oral pill taken twice a day for the treatment of CF in people ages 6 and older with the G551D mutation.

In people with this mutation, Kalydeco helps improve lung function and lower sweat chloride levels and helps patients gain weight — all key indicators that the drug is working.

Kalydeco was developed by Vertex Pharmaceuticals Inc. with significant scientific, clinical and financial support from the CF Foundation, including a $75 million investment.

The drug was formerly called VX-770 and is also known by its generic name, ivacaftor.
 
The U.S. Food and Drug Administration (FDA) approved Kalydeco for people with the G551D mutation ages 6 and older in January 2012.

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How does Kalydeco work?

In people with the G551D mutation of CF ages 6 and older, Kalydeco helps improve the function of a defective protein, called CFTR.

In people with this mutation, the defective protein moves to the right place at the surface of the cell but does not function correctly. Instead, it acts like a locked gate, preventing the proper flow of salt and fluids in and out of the cell.

Kalydeco helps unlock that gate and restore the function of the CFTR protein. This allows for a proper flow of salt and fluids on the surface of the lungs. This helps to thin the thick, sticky mucus caused by CF that builds up in the lungs.

Kalydeco marks a breakthrough in CF treatment because it is the first drug to address the underlying cause of cystic fibrosis. All other therapies available address the symptoms of the disease, not the underlying cause.

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How can I or my child obtain Kalydeco?

Kalydeco is available by prescription for people with the G551D mutation ages 6 and older. Consult with your doctor or CF care center about whether Kalydeco is the right therapy for you or your child.

To learn more about Kalydeco, contact Vertex directly at 1-877-752-5933 or visit www.kalydeco.com.

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

For more information on obtaining Kalydeco, contact Vertex directly at 1-877-752-5933 or visit www.kalydeco.com.

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Is Kalydeco only for those with the G551D mutation?

The FDA has approved Kalydeco only for people with the G551D mutation of CF ages 6 and older.

More studies are needed to help determine whether people with other CF mutations might be eligible for Kalydeco at a later date.

Vertex plans to begin clinical trials in 2012 studying Kalydeco in CF patient groups that were not evaluated in earlier studies of the drug, including children as young as age 2 with the G551D mutation.

Kalydeco has also been studied in a Phase 2 clinical trial in combination with another oral medication, VX-809, in people with the Delta F508 mutation of CF. In addition, Vertex is enrolling people with two copies of the Delta F508 mutation for a Phase 2 study of Kalydeco with VX-661, a CF drug in development, in people with two copies of the Delta F508 mutation.

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Is Kalydeco a cure for CF?

People with the G551D mutation — about 4 percent of the CF population in the United States — will benefit directly from the drug, but it is too early to say whether Kalydeco will be an actual cure for them.

Kalydeco has demonstrated it can markedly improve lung function, lower sweat chloride levels and help patients gain weight.

It marks a breakthrough in CF treatment because it is the first drug to address the underlying cause of the disease.

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If I start taking Kalydeco, can I stop taking my other CF medications?

Before starting Kalydeco, talk to your CF doctor about how the drug fits into your treatment regimen.

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How many people with CF have the G551D mutation?

About 4 percent of people with CF in the United States have the G551D mutation.

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

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

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

To learn more about the MAP, talk with your CF doctor or care center.

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Will Kalydeco help people with other CF mutations? 

More research will be needed to answer this question. Laboratory studies suggest that, in addition to those with the G551D mutation, people with certain other CF mutations may respond to Kalydeco.

Vertex plans to begin clinical trials in 2012 studying Kalydeco in more CF patient groups, including:

  • Children ages 2 to 5 with the G551D mutation.
  • People with gating mutations of CF besides G551D.
  • People with at least one copy of the R117H mutation.

In gating mutations like G551D, the defective protein in CF moves to its proper place at the cell surface but does not function correctly. The CFTR protein instead acts like a locked gate, preventing the proper flow of salt and fluid in and out of the cell. Kalydeco aims to unlock that gate.

The R117H mutation, known as a conductance mutation, causes abnormal function of the CFTR protein at the cell surface.

Kalydeco is designed to improve the function of the defective CFTR protein once it is at the surface of the cell. For this reason, it is possible that people with gating mutations besides G551D and those with the R117H mutation may benefit from Kalydeco.

Kalydeco has also been studied in a Phase 2 clinical trial in combination with another oral medication, VX-809, in people with the Delta F508 mutation of CF. In addition, Vertex is enrolling people with two copies of the Delta F508 mutation for a Phase 2 study of Kalydeco with VX-661, a CF drug in development.

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What is being done to create other drugs targeting the underlying cause of CF that might help more people? 

The CF Foundation is committed to its mission to find a cure for CF for all people with the disease as soon as possible.

Among the research priorities of the CF Foundation is developing a therapy that targets the underlying cause of the disease in people with the most common mutation of CF, Delta F508.

Progress toward this goal includes a Phase 2 clinical trial that tested Kalydeco in combination with VX-809, a potential CF therapy, in people with at least one copy of Delta F508.

In people with this mutation, the defective CFTR protein does not move to its proper place at the cell surface. VX-809 is designed to move CFTR to the cell surface; Kalydeco helps improve the protein’s function once it reaches the cell surface.

Results from the second part of this Phase 2 trial showed significant improvements in lung function in people with two copies of the Delta F508 mutation who received the combination treatment. Vertex plans to begin a pivotal trial of Kalydeco and VX-809 in people with two copies of Delta F508 in early 2013.

Vertex is now studying Kalydeco in a Phase 2 clinical trial with another therapy in development, VX-661, in people with two copies of the Delta F508 mutation.

In addition, the CF Foundation has significantly expanded its research investments with other leading pharmaceutical companies, including Genzyme and Pfizer, to speed the discovery and development of similar drugs to repair the faulty CFTR protein in people with the Delta F508 mutation of CF.

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

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What was the CF Foundation’s role in the development of Kalydeco?

Kalydeco was discovered in a collaboration between Vertex and the Cystic Fibrosis Foundation that began more than a decade ago. The Foundation provided significant scientific, clinical and financial support throughout the development process, including a $75 million investment.

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Updated 5/3/13

The Cystic Fibrosis Foundation is an accredited charity of the Better Business Bureau.