background

People with CF pictured may 
or may not be taking ORKAMBI.

How ORKAMBI® Works

Everyone’s cystic fibrosis (CF) is different, but ultimately, it still works beneath the surface. Since CF is working beneath the surface, the treatment should be, too. With ORKAMBI, you can treat the underlying cause of CF in people age 2 years or older with 2 copies of the F508del-CFTR mutation.


The underlying cause of CF

CF is caused by cystic fibrosis transmembrane conductance regulator (CFTR) protein defects. A mutation in the genes of a person with CF may make defective CFTR proteins that:

  • Don't open correctly
  • Don't get to the cell surface, where they are normally located

A person with CF may make CFTR proteins that have one or both of these defects.

Because of these defects, chloride ions cannot flow freely into or out of the cells as they should, leading to an imbalance of salt and water. This can lead to thick, sticky mucus in the lungs.

See how ORKAMBI works

Watch a video to see how ORKAMBI works

See how ORKAMBI targets the underlying cause.

ORKAMBI targets the underlying cause

ORKAMBI is made up of lumacaftor and ivacaftor, which work on certain defects of the CFTR protein at the cellular level.

 

the cell of someone with CF without ORKAMBI® (lumacaftor/ivacaftor) and with ORKAMBI, showing how Ivacaftor and Lumacaftor work together
the cell of someone with CF without ORKAMBI® (lumacaftor/ivacaftor) and with ORKAMBI, showing how Ivacaftor and Lumacaftor work together
the cell of someone with CF without ORKAMBI and with ORKAMBI, showing how Ivacaftor and Lumacaftor work together

Together, lumacaftor and ivacaftor may help more chloride ions to pass into and out of the cells—helping keep a balance of salt and water in certain organs, such as the lungs.

What is known about how ORKAMBI works was learned from studies conducted in a laboratory. Keep in mind that laboratory studies do not always match how these medicines work in a person. If you have any questions about your treatment, please speak with your healthcare provider.