Cystic fibrosis (CF) patients can now get a “life-transforming” treatment on the NHS in England.
Nine in 10 people with the genetic condition – more than 7,000 in England – could benefit from the three-drug combination called Kaftrio, say experts.
NHS boss Sir Simon Stevens said a landmark deal had been made with Vertex Pharmaceuticals which makes the drug.
The deal does not apply to the NHS in Scotland, Wales and Northern Ireland.
Children under the age of 12 will not be able to access the treatment either because the European licence does not permit this.
Alexandra Andrews, 45 and from Nottingham, who was able to start the treatment on compassionate grounds ahead of the announcement, called it “amazing”.
She has been taking it twice a day for about eight weeks.
“It’s been my mini-miracle. It’s improved my quality of life no end. I can actually do more of the little things in life.
“I’m not completely off oxygen but it’s reduced how much I need. And I’m not coughing all the time and stuck in bed. I’ve got my energy back. I’ve even been able to mow the lawn.”
Cystic fibrosis can affect individuals differently, but the symptoms are caused by a build-up of thick sticky mucus in the lungs, digestive system and other organs.
Some people will have milder disease than others.
Alexandra has the most common type of CF, caused by two copies of the faulty gene involved.
CF can be diagnosed by the heel-prick test that all new-born babies in the UK receive.
The therapy combines three drugs – ivacaftor, tezacaftor and elexacaftor – to tackle the underlying causes of the disease, by helping the lungs work effectively.
Two of these drugs are already available in the UK on the NHS under different brand names – Orkambi (which is a combination of lumacaftor and ivacaftor) and Symkevi (ivacaftero and tezacaftor).
The deal – supported by the drugs watchdog the National Institute for Health and Care Excellence – will last for four years and allow for further data to be collected to better understand the benefit to patients.
David Ramsden from the Cystic Fibrosis Trust said: “This is fantastic news that a deal has been done between NHS England and Vertex Pharmaceuticals and Kaftrio will now be available to thousands of people across England in the coming weeks.
“This will truly save lives. This is a great day, but we know that there is more to do and we will not stop until everyone with cystic fibrosis across the UK has access to life-saving drugs.”