Portsmouth mum ecstatic after life-changing cystic fibrosis drug is approved for her daughter

A MUM who has campaigned tirelessly for four years – including visiting Downing Street with a petition – was ecstatic after an agreement was reached to allow a life-changing drug to be used on cystic fibrosis sufferers.

By Steve Deeks
Thursday, 24th October 2019, 2:52 pm
Ivy Weir with her mother, Gemma. Picture : Habibur Rahman
Ivy Weir with her mother, Gemma. Picture : Habibur Rahman

Gemma Weir, of Paulsgrove, admitted she never thought the day would come but today it was announced by drug manufacturer Vertex that a deal had been struck with the NHS to license Orkambi in England.

Gemma, whose daughter Ivy has the life-limiting illness which means she has just a 50 per cent chance of living to 31 years of age without the drug, ‘screamed’ with joy when the news came through.

She caught the moment on video, above, as she took to Facebook Live to tell her friends, families and followers the good news. 

The mum said the move will transform her daughter’s life, who will no longer have to live with a ‘horrible life expectancy hanging over her head’.

She told The News: ‘I was in a coffee shop when I heard the news and just screamed and shouted. I’m totally gobsmacked – I’m so happy. 

‘I can’t believe they have finally agreed it. I never thought it would happen. It means Ivy will no longer have to live with a horrible life expectancy hanging over her head and will be able to take a drug that makes her better.

‘We have been campaigning for four years for this day – it’s a very special day. I’m so grateful to Matt Hancock (secretary of state for health and social care). We knew they could get the deal done and now they have.’

The announcement came when the family were on the verge of flying to Argentina for treatment of an alternative drug similar to Orkambi.

Gemma added: ‘It got to the point where we never thought it was going to happen so were preparing to go to Argentina. But now we don’t need to as Ivy will have the best treatment here.’ 

Vertex had been locked in dispute with the NHS over the pricing the drug – said to cost £104,000 per patient per year, which the NHS said was too expensive.

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Patients with cystic fibrosis will now be able to receive the drug within 30 days.

Ludovic Fenaux, senior vice president of Vertex International, said: ‘Today is a significant day for the cystic fibrosis community in England. 

‘This important agreement, reached in collaboration and partnership with NHS England and NICE, will allow more than 5,000 eligible cystic fibrosis patients in England to have access to CFTR modulators to treat the underlying cause of their disease.’

The Cystic Fibrosis Trust posted on Twitter: ‘A deal has been done. Finally, people with cysticfibrosis living in England will have access to Orkambi and Symkevi. We thank the community for their support and campaigning to get access to LifeSavingDrugsNow!’

In addition to England, reimbursement agreements have also recently been announced in Scotland, Australia and Spain.