Kaftrio now approved for 2–5-year-olds
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 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 2–5-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.