Mum of little girl, three, with cystic fibrosis in fight to see wonder drug licenced in UK

Cystic Fibrosis medication awarness. Emma Corr with daughter Harriet, 2

Emma Corr, 37, of Wansbeck Road, Jarrow, said more than 75,000 people had signed online in just five days – almost half the 100,000 target.

If that figure is achieved, rules state the nationally organised petition must go before government.

Cystic Fibrosis medication awarness.
Emma Corr — Cystic Fibrosis medication awarness. Emma Corr

It has been launched in response to several other countries giving the green light for Orkambi to be used to treat children aged six and over, in recent weeks. The drug attacks the root cause of the progressive lung condition rather than only its symptoms.

Mrs Corr, an English teacher, whose daughter Harriet, three, has the condition, said she and other parents were angry it was available overseas but not in the UK.

She said: “We are meant to have one of the world’s great health services, yet Orkambi is still not allowed here.

“This is a drug that actually corrects faults with lungs on a cellular level, it’s make a real difference to people’s lives.

“If this drug was allowed, there would be savings on hospital admissions, existing medication and it would free up space in hospitals.

“People are dying every day because Orkambi is not available in this country, but it really should be and it’s time it was.

“The parents of children with cystic fibrosis are angry and frustrated by this – it’s time the government and the drug company got together and made it affordable.”

The drug is believed to cost about £104,000 a year per patient, and there are estimated to be 10,000 people with the condition in the UK.

Most have the type of cystic fibrosis that Orkambi is designed to treat.

Mrs Corr says if treated, those with cystic fibrosis, a condition where fluid builds up the lungs, can lead full and active lives.

They can also achieve a normal life expectancy, rather than the average of 30.

Orkambi works by helping water and salt pass normally out of the lungs.

NICE, which provides guidance and advice to the Government around health, has said it is aware of the importance of effective treatments but says the cost, relative to its benefits is too high, but that it welcomes further feedback from its manufacturer, Vertex.

The petition can be found at https://petition.parliament.uk/petitions/209455

%EF%BF%BD.