24 August 2020

NHS patients - including here at Royal Papworth Hospital - are set to be among the first in Europe to benefit from a life-changing new treatment for cystic fibrosis. 

Kaftrio, also known as the ‘triple combination therapy’, was given the green light by European regulators on Friday (21 August), setting live a deal struck by NHS England in June to get the drug onto the frontline of patient care as soon as it was licensed. 

The news comes just eight months after a previous deal with Vertex Pharmaceuticals for Symkevi, Orkambi and Kalydeco to also be made available to NHS patients. 

We are absolutely overjoyed at this news

CF is an incurable genetic condition where the lungs become clogged with thick, sticky mucus. This is because people with CF either do not have enough or have ineffective proteins which results in mucus and digestive fluids being too thick. This leads to blockages, inflammation, increased risk of infection and poor digestion. 

Kaftrio treats the underlying causes of CF, by increasing the number of proteins and making them more active, thereby relieving symptoms. 

Dr Uta Hill, Lead Clinician for CF, said: “We are absolutely overjoyed at this amazing news for our patients. We believe that this will mark a game-changer for people with cystic fibrosis as it will dramatically improve their quality of life.

“There will be some patients who may not yet be eligible for such treatments, however there is ongoing research and clinical trials which will ensure that these patients will also benefit from therapies in the future.” 

We hope to be able to prescribe Kaftrio to our eligible patients in the coming weeks

Some people who are already on the treatment have seen life-changing benefits: increased lung function; activity levels up; improved wellness scores; weight gain; and in a handful of instances patients have even managed to be steered away from needing a lung transplant. As with any drug, there are side effects for some who take Kaftrio and not everyone is eligible; it is only used on patients aged over 12.

“The CF team here at Royal Papworth Hospital is committed to getting Kaftrio to all of our eligible patients as quickly as possible,” added Dr Helen Barker, Consultant Respiratory Physician. 

“There is a lot of work to do but please rest assured we are already well under way with the process and although we cannot prescribe immediately, we hope to be able to do so in the coming weeks.”

Read more about the landmark NHS deal (NHS England website)

Find out about Kaftrio, including how it works, its benefits and risks (European Medicines Agency website)

Learn about the CF service at Royal Papworth Hospital