Kaftrio (Trikafta in the US) – Triple combination therapy

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 has formally licensed Kaftrio for use by people with CF who are:

  • 12 years and older who have one copy of the F508del mutation regardless of the other mutation type
  • People with gating (F/G) or residual function (F/RF) mutations now eligible for the triple combination therapy

The MHRA announced approval for the same people with CF in the UK.

Kaftrio is not yet licensed for those under the age of 12 in the UK. The FDA in the US extended the license to 6-11-year olds with cystic fibrosis who have at least one F508del mutation. We are monitoring any progress with Vertex’s planned license extension submission for 6-11 year olds and will update this page accordingly when we have more information.

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.

We have produced a factsheet in November 2020 on the emotional and social impact impacts of Kaftrio, for those who are eligible for the treatment, as well as those who will not be able to benefit from it. Download the factsheet.

When did Kaftrio become available?

Following formal licensing by the European Commission, clinicians across the UK are able to prescribe 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,  this enabled the drug to be prescribed as soon as it was licensed in the UK on 21 August 2021. 

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. 

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. Anyone with one copy of f508del mutation can now access the modulator and we are continuing to motor roll out more widely for younger age groups to ensure everyone that those who could benefit can do so 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.

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 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.

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