A CARLISLE family is planning a huge celebration after news this week that a cystic fibrosis (CF) treatment, described as being “as close to a cure as possible”, will be made available on the NHS.

The Louden family has spent weeks in isolation due to five-year-old Ayda’s condition but 2020 is finally looking up for them as NHS England announced it had made a landmark deal with Vertex Pharmaceuticals for its new triple combination treatment Kaftrio.

Ayda’s dad Dave said the past week had been “incredible” and was already planning a family party for when it’s finally safe to do so.

The 36-year-old campaigned tirelessly for access to another precision medicine, Orkambi, which has drastically improved his daughter’s quality of life since she started the treatment in December.

But he said this new medicine was a “game changer” for Ayda and the majority of CF patients.

“The way we see it, and hope for as a family, Orkambi is the drug that will extend her life to have a husband and kids, Kaftrio is the drug that will allow her to see her kids grow and see her grandchildren,” he said.

While Ayda is currently too young to start Kaftrio, which the European Medicines Agency (EMA) has granted for patients aged 12 and over, trials are already under way for those aged six to 11. Dave hopes it will be available for younger children by the time Ayda reaches her sixth birthday next March.

“We are so lucky she has Orkambi in the meantime to keep her health and body stable until that time, whenever it may be, that she can start Kaftrio,” he added.

NHS England’s landmark deal means clinicians will be able to prescribe Kaftrio to patients - as soon as the licence is granted - in the coming weeks.

Like Orkambi, this treatment - known as Trikafta in the US, where it has been available since October - tackles the underlying cause of the disease by helping the lungs work effectively. It has shown unprecedented results in clinical trials.

While Orkambi can treat 50 per cent of the CF population, Kaftrio will benefit nine out of 10 CF patients - more than 7,000 people.

“Our consultants have been supportive of Orkambi but they have always said this would be a ‘game changer’” said Dave.

“It absolutely outstanding. The clinicians say it’s four to five times more effective than Orkambi which is incredible. They say it will change the way CF is treated.”

In evidence submitted to NICE for its approval process, Dr Keith Brownlee, of the Cystic Fibrosis Trust, said: “The CF community see this treatment as close to a cure as possible. This is the most promising drug in an innovative pipeline that addresses the underlying cause of CF symptoms and is therefore an essential step-change in our progression towards a cure for the condition.

“The results cannot be disputed as not only has it shown to have a tremendous impact on a person with cystic fibrosis’ overall health, but it may have wider financial benefits to the NHS.”