Modulators
Unlike traditional treatments that manage symptoms, cystic fibrosis transmembrane conductance regulator (CFTR) modulators correct the malfunctioning protein made by specific CFTR gene mutations. Learn how these innovative therapies have changed CF care, who can benefit from them, and the latest developments in research and access.
Alyftrek
Alyftrek, made up of vanzacaftor, tezacaftor and deutivacaftor, is the latest CFTR modulator.
Kaftrio
Kaftrio is a triple combination therapy, made up of ivacaftor, tezacaftor and elexacaftor.
-
What are modulators?
Cystic fibrosis is caused by mutations, or errors, in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The mutations mean that the CFTR protein doesn't work properly or isn't made at all.
CFTR modulators are medicines that help fix this protein so it can work better. They're the first type of medicine that treats the root cause of CF, rather than the symptoms.
Different mutations affect the CFTR protein in different ways, so modulators are designed to fix specific problems. This means each medicine only works for people with certain mutations. Unfortunately, not everyone with CF can benefit from modulators.
There are five CFTR modulators available:
-
How do modulators work?
Modulators help the CFTR protein work better so it can do its job of regulating the flow of water and chloride in and out of cells.
When chloride can move more normally, the mucus in the lungs and other organs becomes thinner and less sticky.
There are two types of CFTR modulators: correctors and potentiators. A corrector helps correctly fold the CFTR protein. A potentiator helps open the channel in CFTR to allow chloride out.
The combination therapy modulators combine more than one compound, including a potentiator and a corrector. These drugs work together to impact the production or function of the CFTR protein, or otherwise improve lung function.
-
Are modulators available on the NHS?
Yes – in 2024, after over a decade of campaigning, modulators were permanently approved for use on the NHS across the UK.
-
What's the Trust doing for those who can't benefit from modulators?
Modulator drugs don’t work for everyone, but we won’t stop until everyone with cystic fibrosis has access to the most effective medicines. That's why we’re investing in vital research projects into effective treatments for all, including genetic therapies.
You might also be interested in
How is CF treated?
Thanks to breakthroughs in medication, physiotherapy and other treatments, people with cystic fibrosis (CF) are living longer and healthier lives than ever before.
Physiotherapy
Physiotherapy is a crucial part of the daily treatment regime required to keep people with cystic fibrosis (CF) fit and healthy.
Our Helpline
Our Helpline is available to anyone looking for information or support with any aspect of cystic fibrosis, a listening ear, or just to talk things through.