Kalydeco is one of only two licenced treatments known to tackle the underlying cause of CF rather than the symptoms, and has been shown to increase lung function from an average of 55.4% to 64.1%.
The Trust’s fight for access to Kalydeco, and the other precision medicine Orkambi, is part of Stopping the Clock, the Trust’s campaign to ensure swift access to innovative medications as they are developed and become available.
“I was over the moon”
Jennifer Moreton, aged 20 from Tonyrefail in Wales, will be one of nine people who could now benefit from the extended criteria. She said:
“I was over the moon when I found out that Kalydeco can help people with my specific genotype, as I have quite a rare form of cystic fibrosis. I have heard stories from other people on the drug who have had their lives transformed by it.
“I shield my condition from my daughter Olivia, who is only two. This means I don’t do my medication or physio when she is around. Naturally I worry about not being well enough to look after her properly but my lung function is quite stable so the drug is likely to help keep it that way.
“I was diagnosed with CF at birth but consider myself one of the lucky ones. I know that half of all those with CF who died last year were under 30, so I am already breaking records.
“While there is no cure, I truly see Kalydeco as a ‘wonder drug’ and being allowed to have it fills me with hope, I am so happy about it I can’t even put it into words.”
Putting pressure on government
David Ramsden, Chief Executive of the Cystic Fibrosis Trust, has written an open letter to the governments of England, Northern Ireland and Scotland, where 310, 26 and 56 people respectively affected by the R117H mutation are still waiting for access to the life-saving treatment, calling on them to take urgent action to make the drug available.
Read the letters from David Ramsden to the governments of England, Northern Ireland and Scotland.
The campaign so far
The Trust successfully campaigned for access to Kalydeco for a number of ‘gating mutations’ in 2012 and 2015, which meant that more than 17 people in Wales stood to benefit from the drug. As a result of this extended criteria, that figure has now risen to 26.
According to the 2015 UK CF Registry report, 433 people in the UK have the R117H genotype, which equates to 4% of people with CF in England, 7% in Scotland, 3% in Wales and 7% in Northern Ireland.
Molly Jones, Public Affairs Officer at the Trust, said:
“It’s brilliant news that more people living with cystic fibrosis in Wales are eligible for this breakthrough treatment. It is one of the only medicines available that helps to address the cause of cystic fibrosis.
“We now urge other nations to follow Wales’ lead and make the treatment available to people who could benefit from it, and stop discriminating against rarer forms of cystic fibrosis.
“The Trust will continue to campaign for personalised medicines to be made available to everyone that can benefit from them.”
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