Approval of drug therapy a 'new dawn' for cystic fibrosis care in Ireland

A drug which has the potential to treat the underlying cause of cystic fibrosis for 80% of patients in Ireland has been approved by the HSE.

The Trikafta drug therapy will be made available to eligible patients once it has been approved by the European Medicines Agency.

The triple combination treatment is not currently approved for use in Ireland.

It is the first approved treatment that is effective for people with cystic fibrosis who are aged 12 and over and who have at least one F508del mutation.

90% of patients worldwide and 80%, or 1,000 people, in Ireland are affected by this mutation.

Philip Watt, CEO of Cystic Fibrosis Ireland said the approval was "a most wonderful Christmas present for all those with cystic fibrosis in Ireland".

He said: "The drug therapy ‘Trikafta’ is what many scientists and patients have been waiting for since the basic genetic cause of CF became understood in 1989.

"One clinical trial showed that Trikafta increased lung function by a mean of 14%.

"Research also shows a decrease in exacerbations by up to 60%.

Mr Watt said: "The decision today effectively means Ireland will be the first country in Europe to receive this drug for its patients.

"We have great hope now that the life expectancy of people with cystic fibrosis in Ireland will increase steadily over the next few years and, indeed, Ireland may overtake other countries.

He said it was "a new dawn for CF care in Ireland" and thanked the Minister for Health Simon Harris and the Government for their "care and compassion".

The Minister for Health welcomed the announcement and said it was a "milestone" for people with cystic fibrosis.

He said: "This is incredible news for CF patients and their families and is another important step on the road to improving outcomes for cystic fibrosis patients. "

Trikafta was approved in the US on October 21. The decision from the European Medicines Agency is expected early next year.

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