Orkambi access extended to younger children

Cystic fibrosis treatment had only been available to people aged 12 and over

Children aged between 6 and 11 will now be able to access the cystic fibrosis (CF) drug Orkambi following regulatory approval from the EU.

The Government agreed a deal with Vertex, the maker of Orkambi, in April 2017 to allow State-funded access to the treatment for people aged 12 and over who have CF.

Provision of the drug for younger children was approved by the European Commission on Wednesday.

The original deal agreed between Vertex and the Health Service Executive covered access for children aged 6-11 should approval be granted by the EU, so this will require no further domestic approval.


Clinical studies have shown that the drug can improve lung function, which is adversely affected by CF. Ireland has a disproportionately high incidence of people living with the condition, and Vertex said 3,400 children across Europe between the ages of six and 11 have some form of CF.

In a statement released on Wednesday, the company said the existing reimbursement agreement with the Government would allow for “rapid access” to Orkambi for the younger age cohort.

Minister for Health Simon Harris welcomed news of the EU approval for Orkambi in a tweet, and said access will be widened "very soon".

“Good news today for CF patients that Orkambi will be available to younger children (aged 6-11) too after EU approval. We ensured this age group was included in our deal reached last year so looking forward to younger Irish children having access soon, very soon!”