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 cystic fibrosis patients and families face complicated issues related to getting the care they need. But CF Foundation Compass makes sure that no one has to do it alone.
For many people with cystic fibrosis, dealing with insurance is as much a part of living with the disease as nebulizers and vests. Many people with CF and their families face issues related to getting the care they need, but no one has to do it alone.
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.
Tezacaftor/ivacaftor (Symdeko™) is approved for individuals with two copies of the most common cystic fibrosis mutation, F508del, as well as for individuals who have a single copy of one of 26 specified mutations -- regardless of their other mutation.
Published on February 12, 2018
The U.S. Food and Drug Administration (FDA) today approved a new drug that treats the underlying cause of cystic fibrosis, expanding the number of people with CF who could benefit from cystic fibrosis transmembrane conductance regulator (CFTR) modulators. This approval paves the way for new, more effective triple combination therapies (treatments consisting of three different modulators, including tezacaftor) scheduled to begin late stage clinical trials in the next several months.
Tezacaftor/ivacaftor (Symdeko™) is approved for people with CF ages 12 and older who have two copies of the most common CF mutation, F508del, providing another option for people in this population, particularly individuals who could not tolerate a similar drug, lumacaftor/ivacaftor (Orkambi®). Around 46 percent of the more than 30,000 people with CF in the U.S. have two copies of F508del.
The drug also was approved for people with CF ages 12 and older who have a copy of one of 26 specified mutations (listed below) of the key protein involved in cystic fibrosis, CFTR. These mutations were selected based on a combination of clinical and laboratory data.
“Today's approval is exciting news for the cystic fibrosis community and a big step forward in our ongoing efforts to find new and better treatments to address the underlying cause of the disease,” said Preston W. Campbell, III, M.D., president and CEO of the CF Foundation. “We are optimistic that next-generation CFTR modulators that build on this advance could bring transformative treatments to nearly 90 percent of people with CF, and we remain committed to finding effective new treatments for every individual living with the disease.”
Tezacaftor/ivacaftor is a drug that improves the function of the CFTR protein. Cystic fibrosis occurs when the CFTR protein is either not made correctly, or not made at all.
In late stage clinical trials, people with two copies of the F508del mutation who had taken the tezacaftor/ivacaftor combination improved their lung function by 4 percentage points compared to those taking a placebo. Participants in the studies also experienced improvements in other key measures of the disease, including a 35 percent reduction in exacerbations (a sudden worsening of symptoms that requires treatment) and an increase in the measurement used to assess quality of life.
People with at least one copy of the 26 specified mutations who took tezacaftor/ivacaftor in late-stage clinical trials improved lung function by 6.8 percentage points compared to those taking a placebo.
Critically, people who took tezacaftor/ivacaftor were less likely to experience tightness in the chest and potential drug interactions, which was observed in a subset of patients on lumacaftor/ivacaftor. (Lumacaftor/ivacaftor is approved for use in people with two copies of the F508del mutations in people with cystic fibrosis ages 6 and older.)
“Symdeko is an important addition to the growing CF treatment arsenal and a much-needed option for individuals with the most common CF mutation,” Michael Boyle, M.D., senior vice president for therapeutics development at the CF Foundation. “We are grateful to the hundreds of people with CF who took part in the clinical trials that made this advance possible and to those in the ongoing trials that hold great promise for our community.”
Early studies strongly suggest that triple combination therapies are potentially more effective and could treat significantly more people than previously approved modulators. Additionally, the trials that tested tezacaftor, ivacaftor, and a third modulator therapy showed positive results in individuals with only one F508del mutation, regardless of their second mutation. Two of these potential triple combination therapies will begin clinical trials by mid-2018.
Tezacaftor/ivacaftor was developed by Vertex Pharmaceuticals Inc. with significant clinical, scientific, and funding support from the Cystic Fibrosis Foundation.
People with CF who are eligible for tezacaftor/ivacaftor include people who have two copies of the F508del mutation and people who have a single copy of one of the following mutations in this table:
For additional information, please see the Vertex release.
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