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Ataluren (Translarna) begins assessment journey

NICE starts gathering information on new cystic fibrosis (CF) drug for ‘nonsense’ mutations.

Today, the Cystic Fibrosis Trust submitted evidence to the National Institute for Health and Care Excellence (NICE) to help paint a clearer picture of the population that could be eligible for the new CFTR-modulating therapy ataluren (Translarna®), produced by PTC Therapeutics. The information submitted by the Trust will help the national body decide the most appropriate method of assessing the treatment.

Ataluren is a new medicine for cystic fibrosis (CF) that targets the basic genetic defect in patients with at least one ‘nonsense’ CFTR mutation. These mutations stop or significantly limit any functional CFTR protein being created. This malfunction means that cells within many vital organs, including the lungs, digestive system and pancreas, do not transport salt chloride effectively. These organs become clogged with thick, sticky mucus that limits their proper function.

Ataluren aims to help the body produce more CFTR protein and reduce this build-up of mucus. The drug has been tested internationally in people with over the age of six who have cystic fibrosis caused by a nonsense mutation.

Clinical trials have demonstrated that the drug interacts with aminoglycoside treatments, such as tobramycin, and that they should not be used together.

Data from the UK CF Registry shows that 499 people in England could be eligible for the treatment, 34 in Wales, 84 in Scotland, and 39 in Northern Ireland.

Nick Medhurst, Policy Manager at the Cystic Fibrosis Trust said: “It is very important that new therapies for cystic fibrosis get a full and fair hearing relating to their use in the NHS and we are very pleased that NICE have begun to gather evidence to help that happen as quickly as possible.

“The challenge remains that many new drugs for cystic fibrosis will only show their true value to the NHS over time and that is why we are working with all stakeholders to investigate ways we can use data collected by the UK CF Registry to support the NHS to make confident investments in exciting new treatments that could be beneficial to people with cystic fibrosis.”

The Trust will continue to share updates on this assessment process.