NHS Expands Access to Cystic Fibrosis Triple Therapy
NHS announces major expansion of cystic fibrosis treatment access.
Hundreds more children and adults will now be eligible for life-changing triple therapy, thanks to an agreement between the NHS and the manufacturer. This decision brings the UK in line with European peers and follows growing clinical demand.
What’s happening?
NHS England is expanding access to a triple combination therapy for cystic fibrosis (CF), making it available to hundreds more children and adults. This move will allow eligible patients aged 2 and above to benefit from a drug regimen that has already shown significant improvements in lung function, infection risk, and overall quality of life.
The treatment: ELX/TEZ/IVA (Kaftrio)
Known commercially as Kaftrio, the therapy combines elexacaftor, tezacaftor, and ivacaftor. It addresses the underlying defect in CF, rather than just symptoms, and has become a cornerstone of modern CF care for eligible genotypes.
Previously available to patients aged 6 and older, the therapy is now being extended to younger age groups following licensing changes and real-world evidence supporting its efficacy and safety.
Who benefits?
Children as young as 2 years old are now eligible, provided they have the appropriate gene mutation. This update is expected to benefit hundreds of families in England, allowing earlier intervention and potentially improving long-term outcomes.
What changed?
The rollout follows a pricing and access agreement between NHS England and Vertex Pharmaceuticals, the manufacturer. This follows MHRA licensing and NICE endorsement of the drug’s clinical value.
This brings the UK closer to other European countries where younger age groups have already been accessing this therapy.
Why it matters
- Earlier treatment can mean better lung health and fewer complications.
- Broader access closes the equity gap for younger patients.
- Alignment with Europe ensures NHS patients are not left behind.
The expansion reflects a broader commitment under the NHS 10 Year Plan to provide early, life-improving interventions for rare and chronic conditions.
Source
NHS England. NHS to roll out new triple combination therapy for hundreds of children and adults with cystic fibrosis. 17 July 2025. https://www.england.nhs.uk/2025/07/nhs-to-roll-out-new-triple-combination-therapy-for-hundreds-of-children-and-adults-with-cystic-fibrosis/