Wonder drug approved for 1500 children with cystic fibrosis

Wonder drug approved for 1500 children with cystic fibrosis

We use your sign-up to provide content in ways you’ve consented to and to improve our understanding of you. This may include adverts from us and 3rd parties based on our understanding. You can unsubscribe at any time. More info

The Daily Express has fought since 2019 for the availability of US firm Vertex’s medication, hailed as “almost a cure” for the condition.

In 2020, the NHS unveiled a landmark Kaftrio deal but, until now, only those aged 12 and over could take it.

But yesterday it was finally approved for children across the UK aged six to 11, meaning 1,500 primary school youngsters will soon be on the life-extending drug.

Out of that number, around 400 children will be taking their first CF treatment, having not been able to take Vertex’s earlier drugs Orkambi and Symkevi. For most families, the shock announcement was the culmination of years of fighting and waiting.

Overwhelmed Marta Kiszka, 35, of Canterbury, Kent – mother of Darcie, six – said: “We’ve been waiting for that for the last six years.

“This is like the feeling of being on the edge of something huge. Something game-changing. It will save her life.”

Alison Fare, 40, of Conwy, North Wales – mother to CF-suffering daughters Imogen, nine, and Annabelle, six – said: “When Imogen was born in 2012 there were no CF drugs and at diagnosis I felt her future looked very bleak and scary.

“Fast forward to 2022 and, after already trying Orkambi and Symkevi, they can soon be on the real icing on the cake, Kaftrio.

“Now I look forward to their future with less fear and dread and more with hope.”

There are around 10,600 Britons with CF, which destroys the lungs with sticky mucus. But 90 per cent can thrive if given the miracle pill.

Desperate families have been waiting for the Medicines and Healthcare products Regulatory Agency to approve it for younger children.

Yesterday, they finally gave it the green light, as did the European Medicines Agency, who approve Northern Ireland’s drugs.

What is happening where you live? Find out by adding your postcode or visit InYourArea

David Ramsden, the chief executive of the Cystic Fibrosis Trust, said: “This is another hugely significant moment for people with CF.

“This is a victory the CF community fought long and hard for and families will be celebrating today, and I am celebrating with them.”

The Daily Express and the CF Trust have now vowed to fight for the 10 per cent of sufferers with rare genes who cannot take Kaftrio.

Mr Ramsden added: “The Trust won’t stop working, so everyone has access to life-changing treatments.”


Young sufferers now have their whole life ahead of them, says CARLIE PLEASANT

When campaigning for our life saving drugs, the younger generation of CF sufferers were in the front of my mind.

I was determined these children would not have to experience what I had to during my childhood.

So today I am overcome with emotion. I’m excited for young sufferers who now have their whole life ahead of them.

I’m elated for their families who finally have tangible hope at their fingertips.

Kaftrio has been life changing for me. Until starting this wonder drug, fighting a winter cold is something I’ve never been able to do without intravenous antibiotics.

Yet I’ve now been hospital admission free for 18 months. I’ve been transformed both physically and mentally.

Nowadays I often hear from many CF parents who look to older CF patients like myself for reassurance.

But it’s wonderful these young children will now be given the pills to prevent their lung function from dropping, prevent hospital stays and gruelling treatment plans.

And the parents will have the reassurance that their children are receiving the most ground-breaking medicine currently available.

Source: Read Full Article