Read about Vertex Pharmaceuticals' triple combination therapy Kaftrio (known as Trikafta in the United States), which combines ivacaftor, tezacaftor and elexacaftor, and find out how it could help to treat people with cystic fibrosis (CF).
What is Kaftrio?
Kaftrio is a 'triple-combination therapy' made up of three different compounds, tezacaftor and ivacaftor (which together make up Symkevi) combined with elexacaftor.
Why is the drug called Kaftrio?
Trikafta is the US name for Kaftrio, whereas the drug has the brand name Kaftrio in Europe. The two names refer to the same drug.
Who is currently eligible for Kaftrio?
The European Commission and the MHRA have formally licensed Kaftrio for use by people with CF who:
- are aged over 6
- have two copies of the F508del mutation or one copy of F508del and any other mutation.
The various drug regulatory bodies across the world take different approaches to how they interpret evidence on the safety and clinical effectiveness of new drugs and ultimately what licence indication should be granted. In the UK, the MHRA is the regulator which makes these decisions.
There may be other reasons why someone is not able to benefit from Kaftrio. We have a factsheet on the complex and individual experiences around Kaftrio, for those who are taking it, as well as those who are not. Download the factsheet.
When did Kaftrio become available?
Kaftrio was first made available to UK patients in August 2020. Since then, the license has expanded to include younger age groups and a wider range of Genotypes.
NHS England announced on 30 June 2020 that a deal had been agreed for Kaftrio. This was followed by similar deals in Wales on 22 July, in Northern Ireland on 30 July and in Scotland on 4 August, this enabled the drug to be prescribed as soon as it was licensed in the UK on 21 August 2020 for patients aged 12 and older with one F508del mutation and one minimal function mutation, or two F508del mutations in the CFTR (cystic fibrosis transmembrane conductance regulator) gene.
The deal with NHS England included 'tag along rights' for the other devolved nations that paved the way for similar deals across the UK and the Crown Dependencies of Jersey, Guernsey and the Isle of Man.
The Trust has worked closely with the UK CF Medical Association to support roll-out and ensure everyone can start Kaftrio as fast as clinically possible.
On 11 January 2022, the European Commission and MHRA approved Kaftrio for eligible children aged 6–11 in England, Scotland, Wales and Northern Ireland.
What effect does Kaftrio have?
In clinical trials for Kaftrio, people with two copies of the F508del mutation had a 10% increase in lung function compared to treatment with Symkevi, and people with a single copy of F508del had more than a 14% increase in lung function compared to treatment with the placebo. Find out more on our FAQ page.
What are we doing to support access to Kaftrio?
We are working with all key stakeholders to support access to everyone in the UK who could benefit.
We are working with the UK Cystic Fibrosis Medical Association to support the roll-out to ensure everyone can start Kaftrio as fast as clinically possible.
We also host and manage the UK CF Registry, which will continue its valuable work in monitoring the effectiveness of this drug, among many others.
Are there other therapies like Kaftrio being developed?
It is difficult knowing that not everyone will stand to benefit from Kaftrio or the other currently licensed modulators.
AbbVie and Vertex Pharmaceuticals are developing other potential triple combination therapies.
Through our Clinical Trials Accelerator Platform, we are actively working to support the ongoing HIT-CF Europe research project which aims to provide better treatments for people with rare CF mutations who do not benefit from Kaftrio.
For some people with CF, transplant remains the only option to prolong their lives. We are working hard to make sure more people with CF are able to have a successful transplant, and to improve support offered to those following on from their transplant.
The Trust is fully committed to finding effective treatment for everyone with cystic fibrosis. You can find out more about our research that leaves no one behind and donate to support this vital work.
How long will it take for my child to get Kaftrio now it has been approved for 6-11-year-olds?
We are hopeful that in the coming weeks more children will get access to Kaftrio. Tests may need to be done before your child begins the medication and we encourage you to speak to your child’s CF Team to get a more accurate timeframe.
Will children under 6 be able to access Kaftrio?
A small clinical trial for Kaftrio in ages 2–5-year-olds is currently taking place. You can find out more about the trial on our Clinical Trials Tracker. We’ll continue to work hard so that people with CF of all ages and mutations have access to the best medicines.
What are you doing for people who can’t take this medication?
Kaftrio won’t work for everyone. And we won’t stop until everyone with CF can access the treatments and medicines they need. We will continue to invest money in life-saving research to find treatments for all. Find out more about some of the research underway to help those who don’t benefit from CFTR modulators.
Learn more about Kaftrio in our Life-saving drugs FAQs.
Fighting for life-saving drugs
Find out more about our campaign to ensure that people with CF across the UK can have access to innovative precision medicines.
Find out what's been happening in our campaign for life-saving drugs since it began in 2015.