A life-extending drug for cystic fibrosis will be available on the NHS in England, health bosses say.
NHS England reached a deal with Orkambi manufacturers Vertex Pharmaceuticals after months of talks. Patients should be able to get the drug within 30 days.
The drug improves lung function and reduces breathing difficulties and can be given to children as young as two.
The firm wanted to charge £100,000 per patient per year but a compromise has been reached in a confidential deal.
It is understood to involve significantly less than the sum originally asked for.
Two other drugs made by Vertex – Symkevi and Kalydeco – will be made available as part of the deal.
These also treat cystic fibrosis symptoms. Symkevi is restricted to over 12-year-olds, while Kalydeco can be used from 12 months.
The treatments do not work for all patients with cystic fibrosis – only those with certain mutations.
It is estimated about half of the 10,000 patients in the UK will benefit from these drugs.
Cystic fibrosis is a life-shortening genetic condition that can cause fatal lung damage.
Only about half of those with the condition live to the age of 32.
‘Best present ever’
Patients and campaigners have expressed delight at the announcement.
Christina Walker said it meant her son, Luis, eight, should be on it by Christmas, calling it the “best present ever”.
“I can’t stop smiling. I’m overwhelmingly happy. It’s absolutely wonderful,” she said.
David Louden, from Carlisle, said the decision would make a “huge difference” to his daughter, Ayda, who has cystic fibrosis.
He said the battle to get it had been “demoralising”.
“You could see this drug with all its benefits that was just hanging there in the balance, dangling like a carrot in front of you but you couldn’t access it.”
‘Good for patients and taxpayers’
The deal comes after the Scottish government reached an agreement with the manufacturers last month.
Under the terms of the agreement both Wales and Northern Ireland can now access the drug for the same price as NHS England, but there has been no announcement over whether they will.
NHS England chief executive Simon Stevens said the deal was “good for patients and fair to British taxpayers”.
Health Secretary Matt Hancock described it as “wonderful news”.
David Ramsden, of the Cystic Fibrosis Trust, said: “This is a very special day and I want to thank people with cystic fibrosis, their families and everyone who has been part of this campaign for their persistence and determination to keep on fighting.”
He also said there could be more good news in the pipeline as a new therapy Trikafta, which 90% of people with the condition could benefit from, was getting close to be licensed for use.