NHS patients across Cambridgeshire will be amongst the first to benefit from a new “life-changing” drug to treat cystic fibrosis (CF).
The European Medicines Agency granted a license last month for the usage of the “ground-breaking” Kaftrio which will be available at Royal Papworth Hospital.
Kaftrio – known also as the ‘triple combination therapy’ – was given the green light, setting live a deal struck by NHS England in June to get the drug onto the frontline.
The Cambridge hospital is the specialist hospital for respiratory diseases in the East of England. It treats more than 350 adult CF patients from across East Anglia.
CF is an incurable genetic condition where the lungs become clogged with thick, sticky mucus.
This is because people with CF either do not have enough or have ineffective proteins which results in mucus and digestive fluids being too thick.
This leads to blockages, inflammation, increased risk of infection and poor digestion.
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Kaftrio treats the underlying causes of CF, by increasing the number of proteins and making them more active, thereby relieving symptoms.
Dr Uta Hill, lead clinician for cystic fibrosis at Royal Papworth Hospital NHS Foundation Trust, said: “We are absolutely overjoyed at this amazing news for our patients.
“We believe that this will mark a game-changer for people with cystic fibrosis as it will dramatically improve their quality of life and prognosis.
“There will be some patients who may not yet be eligible for such treatments, however there is ongoing research and clinical trials which will ensure that these patients will also benefit from therapies in the future.”
Some people who are already on the treatment have seen life-changing benefits: increased lung function; activity levels up; improved wellness scores; weight gain; and in a handful of instances patients have even managed to be steered away from needing a lung transplant.
As with any drug, there are side effects for some who take Kaftrio and not everyone is eligible; it is only used on patients aged over 12.
Dr Helen Barker, consultant respiratory physician, said: “The CF team here at Royal Papworth Hospital is committed to getting Kaftrio to all of our eligible patients across the East of England as quickly as possible.
“There is a lot of work to do but please rest assured we are already well under way with the process and although we cannot prescribe immediately, we hope to be able to do so in the coming weeks.” Read more about the landmark NHS deal.”