New drug for cystic fibrosis sufferers is 'the most wonderful Christmas present'

The first drug to treat the underlying cause of cystic fibrosis (CF) for around 80pc of people with cystic fibrosis in Ireland has been approved by the HSE.

Philip Watt, CEO of Cystic Fibrosis Ireland, said: “This is a most wonderful Christmas present for all those with cystic fibrosis in Ireland.

“The drug therapy ‘Trikafta’ is what many scientists and patients have been waiting for since the basic genetic cause of cystic fibrosis became understood in 1989.”

Trikafta is the first approved treatment that is effective for CF patients age 12 years and over with at least one F508del mutation, which affects 90pc of the population with the disease worldwide and 80pc of the cystic fibrosis population in Ireland, or about 1,000 people.

Trikafta was approved in the US on 21 October 2019, some five months ahead of schedule.

All patients in Ireland with cystic fibrosis who are aged 12 years and over and who have at least one copy of the F508del gene mutation will get access to Trikafta as soon as it is approved by the European Medicines Agency (decision expected in the first quarter of 2020).

Mr Watt added: “This means that all those who can benefit from Trikafta aged 12 and over will have access to this drug. It is likely that most of those on existing CF drugs such as Orkambi will transfer over to Trikafta depending on the advice of their clinician.”

One clinical trial showed that Trikafta increased lung function by a mean of 14 pc,” Mr Watt said.

“Research also shows a decrease in exacerbations by up to 60pc. This is a new dawn for cystic fibrosis care in Ireland and we thank Minister for Health Simon Harris and the Irish Government for their care and compassion. 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.”

Source: Read Full Article