A "life-changing" treatment for patients with Cystic fibrosis (CF) will now be available on the NHS.
The NHS has made a deal with Vertex Pharmaceuticals to make the drug available to patients in England.
It will not be available to children under 12 due to licensing regulations.
But the NHS in Scotland, Wales and Northern Ireland could soon opt to make the drug combination available there too.
English CF patients will become some of the first in Europe to benefit from a new “truly life-transforming” treatment, health service chief executive Sir Simon Stevens said.
“This is a potentially transformational treatment for cystic fibrosis,” he said.
“The triple therapy is the latest improvement in care which will help thousands of people with CF lead longer, healthier lives and shows once again that where drug companies are prepared to work flexibly with the NHS we can deliver life-changing medicines.”
Three in five patients with the deadly genetic condition could benefit from the ‘triple combination’ treatment.
This means nine out of 10 patients with cystic fibrosis – more than 7,000 people – will now benefit from a therapy which tackles the underlying causes of the disease by helping the lungs work effectively.
The landmark deal, negotiated ahead of a licence being granted by the European Medicines Agency, means that clinicians will be free to start prescribing the triple treatment to English patients as soon as the licence is finally granted, in the coming weeks.
As many as 300 patients with rare mutations will also be able to benefit from the range of treatments produced by Vertex Pharmaceuticals.
The deal is welcome news for patients with cystic fibrosis who have been advised to shield at home during the COVID-19 pandemic.
Alexandra Andrews, 45, from Nottingham, has been taking the drug since May 2020.
“It’s been amazing,” she said. “It’s improved my quality of life no end. I can actually do more of the little things in life. I can exercise again and I feel healthier.
“This drug has made a massive difference and it’s helped me to feel better mentally too.
“I can now have a conversation without feeling out of breath, without struggling to breath. It’s like a miracle drug and it’s given me a new lease of life."
The new triple combination treatment combines three drugs which perform different functions – ivacaftor, tezacaftor and elexacaftor.
It will further boost NHS patients with cystic fibrosis, who already have full access to other drugs manufactured by Vertex.
The deal follows a campaign by the Cystic Fibrosis Trust calling for access to life-saving drugs.
David Ramsden, chief executive of the Cystic Fibrosis Trust, said: the treatment will “truly save lives”.
He said: “This is a great day, but we know that there is more to do and we will not stop until everyone with CF across the UK has access to life saving drugs.”
Health Secretary Matt Hancock, said he has been “bowled over” by the bravery of families living with cystic fibrosis.
“I’m absolutely thrilled we have now secured a deal for Kaftrio, a cutting-edge medicine which gives hope to families and patients for a longer, healthier and happier future,” he said.
“I will always fight for families and loved ones to have access to the best possible treatments so that everyone has the opportunity to flourish.”
Ludovic Fenaux, senior vice president at Vertex International, said: “I am delighted that eligible patients will be able to benefit from access to triple combination regime in the next few months, should the licence be granted by the European Commission.
“Establishing this agreement, in advance of the medicine being licensed, illustrates the collaboration between Vertex, NHS England and NICE. It shows just what is possible when the biotech industry and the NHS partner together for the benefit of patients.”
