Kaftrio now approved for 2–5-year-olds

News -

The UK medicines regulator, the MHRA, has today extended the licence of the CF drug – Kaftrio (ivacaftor, tezacaftor, elexacaftor) – saying it is safe and effective for eligible children over 2 years old.  

Kaftrio and Kalydeco are taken together by patients as a long-term treatment to manage their symptoms of cystic fibrosis.

The drug manufacturer, Vertex, will now need to make the medicines available to UK CF teams, who can then start to prescribe them. Individual CF teams will organise this process.  

This approval is separate to the ongoing NICE appraisal process. NHS England have confirmed that all eligible children can be confident about their long-term access to these treatments. 

Does this mean that NICE have changed their recommendation about whether Kaftrio is cost-effective?

The NICE appraisal process and regulatory approval of Kaftrio for 2–5-year-olds are two separate and different processes. MHRA approval means the drug is safe and effective for its intended use. The NICE process looks at how well a treatment works, and whether it is cost-effective, to decide whether it should be funded by the NHS.

The NICE appraisal recommendation shared by NICE on 3 November is a first stage, draft recommendation. We are hopeful that there will be a resolution to the uncertainty that announcement has created and we are urging all parties to work together quickly to find a solution.

Eligible children and adults with cystic fibrosis can continue to receive ongoing treatment and be initiated onto treatment, as clinically appropriate.

For more information on the NICE process, please visit our news page, where you can follow the link to submit feedback, and read our FAQs

This is great news and another important step in ensuring access to life changing modulator drugs for all of those with cystic fibrosis who could benefit. I’m also reassured that NHS England has confirmed all children eligible today can be confident about their long-term access to these treatments. Today’s news reinforces the need to ensure that the current NICE process rapidly results in a comprehensive deal to end the uncertainty for all who could benefit in the future.

David Ramsden, Chief Executive at Cystic Fibrosis Trust

Julian Beach, MHRA Interim Executive Director, Healthcare Quality and Access, said: “We have prioritised the assessment of Kaftrio and Kalydeco for this age range in view of the unmet needs of children with cystic fibrosis.

“As with all products, we will keep the safety of Kaftrio and Kalydeco in this age range under close review.”

John Stewart, National Director for Specialised Commissioning at NHS England said: “Following the MHRA approval of ivacaftor/tezacaftor/elexacaftor (Kaftrio®) for children aged 2 – 5 yrs, children as young as two years old with cystic fibrosis will now be eligible to receive the triple therapy if determined to be suitable by their treating clinician.

“We anticipate that as many as 600 children could benefit from this approval under the terms of the existing commercial agreement, and NHS England will publish an updated policy confirming this expansion in access and funding to coincide with stock arriving in England, which is anticipated in a few weeks.

“Patients, families and carers should be assured that NHS CF centres across the country have plans in place to ensure that all eligible children can be initiated on treatment while the NICE review of the CFTR modulators remains ongoing – meaning that all children eligible today can be confident about their long term access to these life-changing treatments”.

What happens next?

Regulatory approval is an important step towards children aged 2–5 with eligible CF mutations receiving Kaftrio. The drug manufacturer will now need to make it available to UK CF teams, who can then start to prescribe Kaftrio for eligible children. It will be up to individual CF teams to organise this process, and this is something they have experience of already from previous roll-outs of modulator therapies to specific age groups.

How long will it take for 25-year-olds to receive Kaftrio?

This depends on lots of factors, but we hope everyone will move as quickly as possible to ensure eligible children can receive Kaftrio quickly.

Will my child be on Kaftrio before the end of current NICE appraisal process?

It’s now up to the manufacturer to get Kaftrio ready for prescribing to eligible children aged 2–5, and then CF teams can make their clinical decisions and go through their processes to prescribe the drug. We don’t know how long that will take but clinical teams have experience of this from previous medicine roll outs.

When they announced MHRA approval for 2–5-year-olds, NHS England also confirmed that all children eligible today can be confident about their long-term access to these treatments whatever the final outcome of the NICE process.

My child has not yet turned 2, but will do in the coming months and I’m worried about what happens if they turn 2 after the NICE appraisal ends, and the final decision is that the medication is not cost-effective.

While the NICE appraisal is ongoing, access to treatment for those starting or already on a treatment will continue. We don’t know exactly what will happen, on what date, at each stage, and this uncertainty may be difficult for some families. NICE have told us that  anyone who needs (based on clinical advice) to move between the drugs covered by the appraisal: Kaftrio, Orkambi and Symkevi will be able to do so at any point in the future.

If you are concerned about this for your child, please do talk with your CF team.

My child is on Orkambi –will they be able to start Kaftrio if not already on it at the end of the NICE process?

NHS England have confirmed to us that  if someone is on Orkambi at the end of the NICE appraisal process, they will still be able to move to Kaftrio, whatever the outcome of the appraisal.  This applies to anyone who needs (based on clinical advice) to move between the drugs covered by the appraisal: Kaftrio, Orkambi and Symkevi at any point in the future. We hope this will be the same in Wales, Scotland and Northern Ireland but we are trying to get confirmation in writing.

If the NICE appraisal’s final recommendation is that the medicines are too expensive, what will this mean for people currently waiting for access (for example, children under 1)?

While the NICE appraisal is ongoing, access to treatment will continue. This will be the case for people starting the treatments at any stage during the appraisal as well as those already taking the treatments. This is not a typical process for NICE to undertake and we do not know exactly what will happen at each stage. The initial recommendation announced by NICE on 3 November 2023 is not a final decision, it’s a step in the wider appraisal process and we’ll be continuing to campaign for access for everyone who could benefit, now and in the future.

You can submit your feedback to the NICE consultation here

If you have questions about this announcement, you can get in touch with our Helpline by emailing helpline@cysticfibrosis.org.uk or calling 0300 373 1000 or 020 3795 2184.

Your donation will make a difference:

Select amount
Select amount