The family of a young girl who has been diagnosed with cystic fibrosis say they have been left disheartened after talks over the availability of a wonderdrug appear to have collapsed.
Emma Corr is one of those campaigning to have Orkambi, made by drugs company Vertex, made available on the NHS.
Everyone thought things were going well and everyone was so positive. Then this news come out the blue.Emma Corr
Her younger daughter Harriet, three, was diagnosed with the condition at five days old, and relies on a variety of drugs and physiotherapy to help keep her alive.
Orkambi corrects faults in the lungs on a cellular level rather than just treating the symptoms of cystic fibrosis (CF), and is available in other countries.
However, despite hopes rising that a deal could be reached between NHS England and the drugs company, an offer to the NHS was knocked back.
Emma, from Wansbeck Road, Jarrow, said: “Everyone thought things were going well and everyone was so positive. Then this news came out the blue.
“It has come as a massive shock to us and the whole CF community can’t believe it. We are just absolutely gutted.
“It’s so disheartening. It was the first time I felt fed up with it all.
“So many people would benefit from this, not just those with Harriet’s mutation. It’s disheartening.”
A spokesman for Vertex said: “In the latest meeting, we made the best offer in the world to NHS England, reflecting our ultimate goal to provide access to all of our medicines for all cystic fibrosis patients as soon as possible. “Our offer also provides budget certainty to NHS England and assurance that patients will have rapid access to advances in medicines soon to come, just like agreements made in other countries.
“We find it outrageous that NHS England does not see a path forward to provide access for thousands of children and young people to the only medicines that treat the underlying cause of CF.
“We call on the Government to intervene to ensure that CF patients in England have access to the medicines that exist now as well as the advances soon to come.
“We stand ready to meet any time, any place, to ensure patient access to these life-changing and transformative medicines.”