Extension of Orkambi to children aged 6-11
The recent decision of the European Medicines Agency (EMA) to extend the CF drug “Orkambi” to children aged 6 to 11 years old, on the basis of clinical research emerging from two Phase III studies (NCT02514473 and NCT01897233), means that this ground breaking drug which had previously only been available to those aged 12 years and older with two copies of the F508del mutation, will now also be available to children aged 6-11 (subject to the advice of their Consultant).
Orkambi treats the most common CF gene alteration/ mutation in Ireland (and the world) that is people who have two copies of the F508del alteration. Orkambi was approved for reimbursement by the Irish Government in April 2017. As part of that agreement, in a unique “pipeline deal”, it was agreed that future extensions of Orkambi or Kalydeco (including this EMA decision to extend Orkambi to children aged 6-11), would be made available to people with CF in Ireland.
Commenting on the extension of Orkambi to children aged 6-11, the Minister for Health Simon Harris said:
“It’s very positive news that Orkambi is now licenced and available for reimbursement in Ireland for CF patients aged 6 to 11.”