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 will be eligible for Kaftrio?
The European Medicines Agency (EMA) have announced a positive opinion for 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.
In addition, clinicians will be able to prescribe Vertex drugs for certain patients with rare mutations that are covered by the U.S. Food & Drug Administration’s licensing decisions.
We are still awaiting the fine details of the deal to understand exactly who is eligible and will update our website and social media pages when this is clear.
When will Kaftrio be available?
Following the EMA’s positive opinion on 26 June 2020, there is now a period of 67 days for scrutiny before the drug can be granted a licence and made available in Europe.
In England, NHS England announced a deal on 30 June 2020 with Vertex to facilitate access from the first day the European licence is granted.
The deal with NHS England includes 'tag along rights' for the other devolved nations and we hope it will pave the way for similar deals across the UK. It is great to see NHS England supporting their colleagues in the devolved administrations, and we are seeking more information and updates about negotiations and access in Wales, Scotland, Northern Ireland, and the crown dependencies of Jersey, Guernsey, and the Isle of Man.
When CF teams can prescribe Kaftrio, it will take time for teams to organise and roll-out Kaftrio. 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.
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. In the first instance, that is seeking more information and updates about negotiations and access in Wales, Scotland, Northern Ireland, and the crown dependencies of Jersey, Guernsey, and the Isle of Man. We are also working to understand the mutations covered in the NHS England deal and what flexibility clinicians will have in prescribing Kaftrio, Symkevi, and Orkambi 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.