"Life-transforming" treatment for cystic fibrosis patients now available on the NHS

Nine in ten people who live with the genetic condition cystic fibrosis - over 7,000 in England - could benefit from a treatment which is now available on the NHS, which includes a three-drug combination called Kaftrio.

NHS boss Sir Simon Stevens recently announced that a landmark deal has been made with the producers of Kaftrio, Vertex Pharmaceuticals.

It has also been said that the NHS in Scotland, Wales and Northern Ireland could opt to follow suit. However, due to European licensing rules children under the age of 12 will not be able to access the treatment.

A number of people living with cystic fibrosis were able to begin prior to the announcement, including 45 year old Alexandra Andrews, from Nottingham, who has called it "amazing". Ms Andrews has been taking it twice a day for about eight weeks and said:

"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."

What is cystic fibrosis?

Cystic fibrosis (or CF) is an inherited condition causing sticky mucus to build up in the lungs and digestive system, which in turn causes lung infections and problems with digesting food. In the UK, CF is often diagnosed by the heel-prick test that all new-born babies receive and affects 1 in every 2,500 babies born. Symptoms of the condition begin in early childhood and can affect individuals differently, but sadly get worse over time, leading to the lungs, digestive system and other organs becoming increasingly damaged. Symptoms include:

• recurring chest infections
• wheezing, coughing, shortness of breath and damage to the airways (bronchiectasis)
• difficulty putting on weight and growing
• jaundice
• diarrhoea, constipation, or large, strong odoured bowl movements 
• a bowel obstruction in newborn babies (meconium ileus), for which surgery may be needed 

Unfortunately there is currently no cure for CF, however a range of treatments can help control symptoms, prevent or reduce complications, and make the condition somewhat easier to live with. Physical activity and various airway clearance techniques may also be recommended to patients to help shift mucus from the lungs.

"Life-transforming" 

The newly available treatment, which has been hailed as "life-transforming" combines three drugs - ivacaftor, tezacaftor and elexacaftor - to combat the underlying causes of cystic fibrosis by helping the lungs work effectively.

Two of these drugs - ivacaftero and tezacaftor - are already available in the UK on the NHS, albeit under different brand names.

The deal between the NHS and Vertex Pharmaceuticals was supported by drugs watchdog the National Institute for Health and Care Excellence and will last for four years, allowing for further data to be collected in order to gain a better understanding of the drug's benefit to CF 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."