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 process is currently paused, and Vertex (who make Kaftrio) can start commercial negotiations with the NHS. While these discussions are happening, 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.


If you have questions about this announcement, you can get in touch with our Helpline by emailing [email protected] or calling 0300 373 1000 or 020 3795 2184.

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