‘Transformative’ cystic fibrosis drugs approved following years of research

Thousands of people living with cystic fibrosis can access the latest treatments in England following a new deal.

Three modulator drugs are being made available on the NHS.

Patients at University Hospital Southampton (UHS) have taken part in pivotal trials that led to their approval.

Southampton researchers have worked with Vertex, the company who developed these drugs, for over a decade.

Cystic fibrosis patient Luke says he’s proud to have trialled drugs that will now change lives on the NHS.

‘Transformative medication’

The National Institute for Health and Care Excellence (NICE) has approved Kaftrio, Symkevi and Orkambi for NHS patients with cystic fibrosis in England. Similar agreements may follow in Wales, Scotland and Northern Ireland.

Around 11,000 people have cystic fibrosis in the UK. It is a genetic condition that causes mucus to clog and damage their lungs, and can significantly shorten their lifespan.

These modulator drugs treat the root cause of the disease, bypassing the genetic error that causes it. They can have a huge impact on patients’ lives.

UHS patients take part in trials at the NIHR Southampton Clinical Research Facility.

Southampton continues to be at the forefront of research in cystic fibrosis. The team recently treated one of the first patients in the world with a gene therapy breathed into the lungs.

Professor Gary Connett, Paediatric Respiratory Consultant at UHS, said:

“UHS has been one of the lead European centres for the clinical trials that were key to getting the licenced approval of all new modulator drugs.

“The NHS has now approved these medicines for continued use for our patients, who were amongst the first in the world to be treated with this new transformative medication.

“People with CF are now enjoying a life more normal, with improved lung function, better nutrition and with a decreased burden of care.”

Proud to have taken part

Luke Southey, a cystic fibrosis patient, says he’s delighted that other patients like him will now be able to benefit from these drugs.

"I feel proud that trialled drugs are now becoming available in the UK,” he says.

“This means the current younger generation of people with cystic fibrosis won't have to go through our older generation’s life of living in hospital on drips. I want them to be happy and lead a healthy life to the best of their ability."

Luke was first diagnosed at age two. Around the age of nine, he was admitted to hospital with a bad lung infection. This meant he needed regular trips to the hospital for intravenous (IV) infusions of medicine.

“After that, it had more of an impact,” his mum Vicki explains, “because we were in hospital every 12 weeks for two weeks of IVs, and that continued right up to when he started the trial at 15. So that was quite a hard six years for him, and his brothers, and all of us really.”

Luke’s condition deteriorated even further – coughing up a lot of blood, losing a lot of weight and struggling with his breathing. He needed to have the IV treatment every four weeks.

Then he was accepted onto a trial for one of these modulators. His mum cried tears of joy when she received the news.

“I actually remember saying ‘it’s better than winning the lottery’,” she says. “I was just absolutely ecstatic that he was getting the chance to start this new drug.”

“To me, it was just another medication to try,” says Luke. “But this one – I didn’t realise how much of a big effect it would have.”

“Last year, I asked my mum ‘if I wasn’t on this medication, where would I be now?’, and I know it sounds horrible to say, but we didn’t think I’d be alive. So it’s had a massive impact.”