Following a continuing campaign by the Cystic Fibrosis Trust to provide people with CF with life-changing treatments, young children in Wales will now have access to Ivacaftor (Kalydeco). Kalydeco is proven to significantly improve lung function, slowing the progression of CF and drastically reducing the amount of time spent in hospital.
The first precision medicine in CF, Kalydeco targets a group of rare mutations that affect about 5% of people with the condition in the UK. The drug allows chloride to move in and out of cells, improving the balance of salt and water in the lungs of people with cystic fibrosis.
Anna Evans, Policy Adviser at the Trust said: “We’re very pleased that young children in Wales will no longer have to wait for access to Kalydeco. We will continue to campaign for access to precision medicines like Kalydeco for the different CF mutations.”
This announcement builds on decisions in Scotland and England to offer the treatment. However, children aged two-to-five in Northern Ireland still do not have access to the treatment.
The Trust is campaigning hard to ensure innovative drugs like Kalydeco are provided to everyone who needs them. This important work includes funding a new Clinical Trials Accelerator Platform to help unblock the pipeline of exciting new treatments that could help improve the lives of people with cystic fibrosis.
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