Delight for Duleek family as cystic fibrosis drug is approved

"It is better than winning the lottery" is how a delighted Duleek parent reacted to the news that his daughter can now avail of the groundbreaking new cystic fibrosis drug Kaftrio.

Neil Dunne, whose eight year old daughter Daisy, has cystic fibrosis, was delighted to hear HSE has now approved the drug for use by children aged 6-11 who previously fell outside the eligibility criteria.

"This is wonderful news. It is a gamechanger for us. Kaftrio is being hailed as a miracle drug. In the past we could only treat the complications and infections, but Kaftrio gets to the root of the problem, it is a massive deal," he said.

Daisy is one of 35 children in Ireland with cystic fibrosis who had previously been excluded from treatment with the drug. The criteria had been based on the mutations involve dint he disease. Its use has now been extended to that group, following lengthy engagements with the drug manufacturer Vertex.

"Last year, we believed she the drug was going to be made available to her, but the drug was pulled from under us when we were told she was one of those excluded.

"Luckily, she has been well, but there were other children in that group who were unwell and regularly hospitalised."

Kaftrio keeps cystic fibrosis patients healthy and symptom free, which in turn increases life expectancy.

Daisy is one of four chlldren of Neil and his wife, Donna and is a pupil of Duleek National School. She has three brothers, none of whom have cystic fibrosis, Ireland's most common genetic disease, which primarily affects the respiratory and digestive systems in children and young adults.

"Daisy has been very well. She is a happy child and understands her condition and knew about this drug, so we are all delighted.

"She has been very well, always has been, but with cystic fibrosis, you never know when something could happen and get worse."

Neil and Donna are all too familiar with the tragic consequences of the disease. "Two of my cousins died from it, one at 18 and the other in her early 40s. My wife's sister died from it at 18," Neil says.

"We knew that Daisy was at high risk of getting the disease as we are both carriers. We knew before she was born here was a one in four chance she would have it and sadly she has".

The new drug is giving great hope as it tackles the underlying causes of the disease, helping the lungs work effectively.

In the past, people who had cystic fibrosis died in childhood, ten years ago the life expectancy was 38 and now the current life expectancy for CF patients born between 2017 and 2021 is 53 years. New drugs and treatments are expected to see that expectancy grow even further.

"This is wonderful news for us, we couldn't be happier. It is better than winning the lottery," says Neil.

Cllr Stephen McKee has warmly welcomed the news that the HSE has approved the Kaftrio drug for those who had been excluded.

"I am delighted that the HSE has now approved the cystic fibrosis drug Kaftrio for use by children aged 6-11 who previously fell outside the eligibility criteria, following robust and lengthy engagements with the drug manufacturer Vertex.

"This drug will greatly improve the lives of the children who can now avail of it so this is a very welcome development. Kaftrio has been a life-changing drug for cystic fibrosis sufferers, and young local cystic fibrosis sufferers who previously could not access the therapy can do so now.

"I recently met with the Minister for Health Stephen Donnelly and I impressed upon him the need to get this drug approved for families that I represent. This is good news for families and the young children with cystic fibrosis who can now access this therapy. It is an important milestone in the treatment of CF in this country," he concluded.