Learn about cystic fibrosis, a genetic disorder that affects the lungs, pancreas, and other organs, and how to treat and live with this chronic disease.
CF is a rare genetic disease found in about 30,000 people in the U.S. If you have CF or are considering testing for it, knowing about the role of genetics in CF can help you make informed decisions about your health care.
If you or your child has just been diagnosed with cystic fibrosis, or your doctor has recommended testing for CF, you may have many questions.
Diagnosing CF is a multistep process. A complete diagnostic evaluation should include a newborn screening, a sweat chloride test, a genetic or carrier test, and a clinical evaluation at a CF Foundation-accredited care center.
Raising a child with cystic fibrosis can bring up many questions because CF affects many aspects of your child’s life. Here you’ll find resources to help you manage your child’s daily needs and find the best possible CF care.
Living with cystic fibrosis comes with many challenges, including medical, social, and financial. By learning more about how you can manage your disease every day, you can ultimately help find a balance between your busy lifestyle and your CF care.
People with CF are living longer, healthier lives than ever before. As an adult with CF, you may reach key milestones you might not have considered. Planning for these life events requires careful thought as you make decisions that may impact your life.
People with cystic fibrosis are living longer and more fulfilling lives, thanks in part to specialized CF care and a range of treatment options.
Cystic Fibrosis Foundation-accredited care centers provide expert care and specialized disease management to people living with cystic fibrosis.
We provide funding for and accredit more than 120 care centers and 53 affiliate programs nationwide. The high quality of specialized care available throughout the care center network has led to the improved length and quality of life for people with CF.
The Cystic Fibrosis Foundation provides standard care guidelines based on the latest research, medical evidence, and consultation with experts on best practices.
As a clinician, you’re critical in helping people with CF maintain their quality of life. We’re committed to helping you partner with patients and their families by providing resources you can use to improve and continue to provide high-quality care.
As part of the Cystic Fibrosis Foundation's mission to help improve the lives of people living with cystic fibrosis, the PSDC initiative taps the CF community to inform key efforts to support the management of daily care.
Your cystic fibrosis care team includes a group of CF health care professionals who partner with you to provide specialized, comprehensive CF care.
Many people living with cystic fibrosis and their families face complicated issues related to getting the care they need. Cystic Fibrosis Foundation Compass makes sure that no one has to do it alone.
CF Foundation Compass is a service that helps people with CF and their families with navigating insurance options, connecting to legal information and experts, finding available financial resources, and tackling other life issues.
CF care team members are paramount in providing highly specialized care to people living with CF. CF Foundation Compass can help by serving as a strategic ally for care teams, so team members can focus on their patients’ care.
CF Foundation Compass can help you navigate insurance, financial, legal, and other issues you are facing. Use this online form to start your conversation with a Compass case manager today.
The Cystic Fibrosis Foundation is the world’s leader in the search for a cure for CF and supports a broad range of research initiatives to tackle the disease from all angles.
The CF Foundation offers a number of resources for learning about clinical trials and treatments that are being developed to improve the treatment of cystic fibrosis.
Our understanding of CF continues to evolve as scientists study what causes the disease and how it affects the body. These insights drive the development of new and better treatments and bring us one step closer to a cure.
Researchers, supported by the CF Foundation, have made tremendous advances to improve the health and quality of life of people with CF. We are committed to providing the tools and resources you need to continuously build upon this work.
CFTR Modulator Therapies
Here's what you need to know about the four CFTR (cystic fibrosis transmembrane conductance regulator) modulators, ivacaftor (Kalydeco®), lumacaftor/ivacaftor (Orkambi®), tezacaftor/ivacaftor (Symdeko®), and elexacaftor/tezacaftor/ivacaftor (Trikafta™).
Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies are designed to correct the malfunctioning protein made by the CFTR gene. Because different mutations cause different defects in the protein, the medications that have been developed so far are effective only in people with specific mutations. There are four CFTR modulators for people with certain CFTR mutations:
More potential CFTR modulators are in development to address the underlying cause of the disease in people with other CF mutations.
The CFTR protein regulates the proper flow of water and chloride in and out of cells lining the lungs and other organs. In people with CF, mutations in the CFTR gene result in either a defective protein being produced or no protein at all. This leads to the buildup of thick, sticky mucus, which can lead to infections in the lungs and damage to the pancreas. It can also lead to problems in other parts of the body.
The following video shows what it looks like when the CFTR protein functions normally in the lungs and what happens when the protein doesn't work.
CFTR modulators have been approved by the U.S. Food and Drug Administration (FDA) for people with the specific CF mutations. The first step in knowing whether you can take these drugs is to know your CF mutations. If you do not know your mutations, specialized genetic tests are available to identify your mutations. The Cystic Fibrosis Foundation's Mutation Analysis Program offers free genetic testing for people with CF who have unknown mutations. If you are interested in enrolling, ask your CF care team.
The newest CFTR modulator elexacaftor/tezacaftor/ivacaftor (Trikafta) is known as the triple combination or triple combo. It combines the next-generation corrector elexacaftor with tezacaftor/ivacaftor. Like lumacaftor and tezacaftor, elexacaftor also helps the F508del-CFTR protein form the right shape so that it can traffic to the cell surface. Because elexacaftor corrects an additional flaw in the formation of the F508del-CFTR protein, including it with tezacaftor/ivacaftor helps the CFTR protein perform better than other modulators for an even greater number of people with CF. The triple combo has been approved for people with CF ages 12 and older who have at least one copy of the F508del mutation.
In people with CF who have gating mutations, the “gate” to the chloride channel at the cell surface is locked. Ivacaftor, a CFTR modulator known as a potentiator, binds to the defective protein at the cell surface and opens the chloride channel (holds the gate open) so that chloride can flow through, regulating the amount of fluids at the surface of the cell. The following video shows how this happens in people who have the G551D gating mutation.
Ivacaftor is approved for people with CF ages 6 months and older who have one of the following mutations:
Lumacaftor/ivacaftor is a combination therapy that is FDA-approved for people with two copies of the F508del mutation. Lumacaftor is a modulator known as a corrector. It helps the F508del-CFTR protein form the right shape, traffic to the cell surface, and stay there longer. But, even with lumacaftor, only about a third of the CFTR protein reaches the cell surface, and those proteins do not open enough to allow chloride to pass through the cell membrane. If a corrector is used in combination with a potentiator -- such as ivacaftor -- to hold the gate on the CFTR protein open, enough chloride can then flow to reduce the symptoms of CF.
The lumacaftor/ivacaftor combination therapy is prescribed for people ages 2 and older who have two copies of the F508del mutation, which is the most common CF mutation. To learn about one person's experience on Orkambi, read, "Orkambi: A Life Sentence."
Tezacaftor, another corrector, acts in the same way that lumacaftor does: It helps the CFTR protein form the right shape, traffic to the cell surface, and stay there longer. The difference between the correctors is that the tezacaftor/ivacaftor combination has been shown to have fewer side effects -- such as chest tightness -- and drug interactions than lumacaftor/ivacaftor. In addition to providing another treatment option for people ages 6 and older with two copies of the F508del mutation, tezacaftor/ivacaftor is also approved for people ages 6 and older with a single copy of one of 26 specified mutations.
To read about the experience of two adults with CF on tezacaftor/ivacaftor, read, “Life on Symdeko Three Months Later” and “How CFTR Modulators Changed My Reality.”
Your CF care team or pharmacist will give you specific instructions for taking CFTR modulators as part of your daily care plan. Instructions for taking CFTR modulators may be different for you than they are for others. Your specific dosage may depend on other medications you're already on, your health needs, and personal circumstances.
The effects of CFTR modulators only last for as long as the medication is in your system. Therefore, you need to take the medication every 12 hours, or as your care team prescribes.
“After 33 years of living with cystic fibrosis, I thought I knew my body pretty well. Since I started on Symdeko®, I have noticed that my body is different, and I need to re-learn how to interpret my symptoms.” -- Aimee Lecointre, adult with CF, from the CF Community Blog
You may experience side effects when you take any medication. When discussing any new medications or changes in dosages for medications you are already taking, be sure to ask your care team about:
Let your care team know if you feel any side effect that bothers you or makes it hard for you to continue taking this medication as prescribed. Your care team can work with you to help you manage side effects or to adjust your treatment plan.
You can learn more about CFTR modulators at DailyMed, which is a service from the National Library of Medicine that provides information about drugs, including dosages and possible side effects.
Reference to any specific product, process, or service does not necessarily constitute or imply its endorsement, recommendation, or favoring by the Cystic Fibrosis Foundation. The appearance of external hyperlinks does not constitute endorsement by the Cystic Fibrosis Foundation of the linked websites, or information, products, or services contained therein.
Information contained on this site does not cover all possible uses, actions, precautions, side effects, or interactions. This site is not intended as a substitute for treatment advice from a medical professional. Consult your doctor before making any changes to your treatment.
FDA-approved drug information is available at dailymed.nlm.nih.gov/dailymed.
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