THOUSANDS of cystic fibrosis patients in England can now benefit from a "truly life-transforming" treatment.
Kaftrio is a triple combination therapy which significantly improves lung function, helping CF suffers to breathe more easily and enhancing their overall quality of life.
From today, patients in England will be among the first in Europe to be prescribed the new treatment, which will benefit nine out of 10 patients with CF – more than 7,000 people.
Dave Louden, from Carlisle, who has long-campaigned for new treatments to be made available on the NHS, said it will save thousands of lives and have a positive impact on the wellbeing of families.
His daughter Ayda, five, suffers from cystic fibrosis, and while she currently is too young to benefit from Kaftrio, he said knowing that it will be available for her in the long-run is a "game-changer" and will help her live a long life.
"It's absolutely brilliant news it’s finally been rubber-stamped today.
"Kaftrio is such an important medicine in the treatment for CF, an absolute game changer. It truly transforms the lives of CF patients for the better.
"It will enable people with CF not only a healthier and longer life, but also open up doors in education, careers and life in general which without Kaftrio would be closed to them.
"The future is now very bright for the majority of CF patients. It’s so encouraging that there has not had to be any relentless campaigning or any delays - which ultimately cost lives - in accessing Kaftrio, as there was with its predecessors.
"Hopefully this will continue for the future generations of precision CF medicines, which will save lives.
"We will always remember those to whom this drug has come too late and will continue to support all research and future medicines until 100 percent of CF patients can be treated, hopefully one day CF can stand for Cure Found. We are not far away with Kaftrio."
Until Ayda can start Kaftrio she has Orkambi, a treatment she started in December, which has drastically improved her quality of life.
In evidence submitted to NICE for its approval process, Dr Keith Brownlee, of the Cystic Fibrosis Trust, said: “The CF community see this treatment as close to a cure as possible... This is the most promising drug in an innovative pipeline that addresses the underlying cause of CF symptoms and is therefore an essential step-change in our progression towards a cure for the condition."
As part of the innovative commercial deal, NHS England also secured equivalent terms for cystic fibrosis patients in Wales, Northern Ireland and Scotland paving the way for full UK access.
As the European licence for the triple combination will only cover patients 12 years and older at this stage, younger children and those over 12 years who are not eligible for Kaftrio will continue to benefit from access to other cystic fibrosis drugs where clinically appropriate, with access to the triple therapy to follow if regulators approve a broader license in the future.
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