'Miracle' CF drug may be available to small children in coming months

Tadhg Griffin with his father Jonathan Griffin in support of neighbours during a protest calling for Government approval for Kaftrio in September 2022. The drug was since approved for children aged 6-11. Picture:  Gareth Chaney/ Collins Photos

The CEO of Cystic Fibrosis Ireland has said he expects a new “miracle” drug for younger cystic fibrosis patients will be approved in a matter of months.

Kaftrio was made available to children aged 6-11 in March of this year following a lengthy campaign.

However, it was recently licensed in America for younger children aged 2-5 — which “stops the damage being done earlier” to CF patients.

The medicine is currently being examined by the European Medicines Agency (EMA). Philip Watt with CF Ireland said he does not see any issues ahead for its approval.

“It is not available to Ireland yet, but it should be made available as soon as possible as long as it's safe to do so," he told the Irish Examiner.

“The EMA is due to make a decision in the next two to three months. So, they will make a decision on their evidence as to whether they approve Kaftrio for children aged two to five years.

“Then, after that, a process will be considered by the HSE as to whether the age extension will be granted in Ireland.

We would expect to know maybe early next year, but the time gap depends on the European agency and the time it takes the HSE to consider it.

“It all depends on if the drug is safe for younger children, and that must be considered. We expect news on this very soon."

Mr Watt added that the effects of the drug have been “very successful” to date, for those to whom it can be prescribed.

“Not everyone can take Kaftrio," he said. “If approved in a younger age group, around 20 patients will benefit from Kaftrio which is hugely helpful.

“We then eventually hope that this drug will be introduced to infants.

 It is a game changer for CF patients and a very important drug and what has been shown so far is, it prevents the damage being done."

Caroline Heffernan from CF Ireland said: “Around 20% of CF patients will never get the drug, and they include people who are outside the genetic realm, while others are outside the age category for the drug and there are those who have had to come off the drug. It just didn't work for them. I am one of those people.

“However, there is a small group of people now living into their 70s with CF, but for anyone with CF there is also the burden of mental health and the burden of treatment.

“We are still not quite there with treatment, it is a positive time, but like the politicians say — a lot done, more to do."

What does Kaftrio do?

Produced by an American laboratory Vertex – Kaftrio is considered a revolutionary treatment for CF patients that can stabilise the disease.

People who have cystic fibrosis have a faulty protein that affects the body's cells, tissues, and the glands that make mucus and sweat.

The tablets reduce the effects of CF and act on the underlying causes of the disease by repairing the defects of a protein CFTR (or cystic fibrosis transmembrane conductance regulator).

Ireland has the highest rate of CF in the world — one 1 in 19 people in the Irish population is a carrier of the defective gene.

Meanwhile the HSE has started a national programme titled 'Implementation of Cystic Fibrosis: A Model of Care for Ir eland' by approving and funding additional cystic fibrosis consultant posts and increased telehealth services.

The statement said these additional consultants will have at least 70% of their time devoted to the care of people with the condition, as specified by the HSE and the National Clinical Programme for Cystic Fibrosis (NCPCF).