THE FIRST DRUG to treat the underlying cause of cystic fibrosis (CF) for around 80% of those with the disease in Ireland has been approved by the HSE. 

The HSE today announced that it has expanded its reimbursement agreement with Vertex Pharmaceuticals. 

As a result, all patients in Ireland with CF who are aged 12 and over and who have at least one copy of the F508del gene mutation will get access to the drug therapy, Trikafta, once it is approved by the European Medicines Agency. 

This is expected to happen in the first quarter of 2020. 

CF, an inherited chronic disease that primarily affects the lungs and digestive system, can greatly impact the lifespan of those with the debilitating condition.

About 1,200 children and adults have CF in Ireland, which has more cases of it per head of population than any other country.

Trikafta was approved in the US on 21 October 2018, some five months ahead of schedule.

Cystic Fibrosis Ireland CEO Philip Watt has said this development 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 on since the basic genetic cause of CF became understood in 1989,” Watt said. 

He added that 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”. 

Health Minister Simon Harris also welcomed the HSE’s announcement. 

“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,” Harris said. 

“This is another milestone and I congratulate everyone who worked to achieve this.”