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 has formally licensed Kaftrio for use by people with CF who:
- are aged over 12
- have two copies of the F508del mutation or one copy of F508del and one copy of a 'minimal function mutation'. The details of which precise genotypes the latter group covers is to be confirmed.
This decision by the EMA is different to the one made by the FDA in America, who allow the drug to be prescribed to people over 12 who have at least one copy of the F508del 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.
When will Kaftrio be available?
Following formal licensing by the European Commission, clinicians across the UK are now able to start prescribing Kaftrio to eligible patients.
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, with Vertex to facilitate access from the first day the European licence is granted.
The deal with NHS England included 'tag along rights' for the other devolved nations that paved the way for similar deals across the UK. We are seeking more information and updates about negotiations and access in the Crown Dependencies of Jersey, Guernsey, and the Isle of Man.
Now CF teams can prescribe Kaftrio, it will take time for teams to organise and roll-out the new drug. The Trust will work closely with the UK CF Medical Association to support roll-out and ensure everyone can start Kaftrio as fast as clinically possible.
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 seeking more information and updates about negotiations and access in the Crown Dependencies of Jersey, Guernsey, and the Isle of Man. We are also working to understand the mutations covered in the deals agreed in England, Wales, Northern Ireland and Scotland deals and what flexibility clinicians will have in prescribing modulators for people with rare or other mutations.
We are also working with the UK Cystic Fibrosis Medical Association to support plans for roll-out to ensure everyone can start Kaftrio as fast as clinically possible.
We also support 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.
At least three companies, including AbbVie, Proteostasis Therapeutics 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 are ineligible for Kaftrio.
The Trust is fully committed to finding effective treatment for everyone with cystic fibrosis. You can read more about our research that leaves no one behind and donate to support this vital work.
Learn more about Kaftrio in our Life-saving drugs FAQs.