EU Approval for Orkambi in Children with CF

EU Approval for Orkambi in Children with Cystic Fibrosis Ages 6-11 with Two Copies of the F508del Mutation 

Cystic Fibrosis Ireland welcomes the decision of the European Medicines Agency (EMA) to extend the Cystic Fibrosis drug ‘Orkambi’ to children aged 6 to 11 years old. Previously the drug had been available to young people and adults aged 12 years and over in Ireland and the rest of the European Union, where approved for reimbursement.

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 (or future drugs that improve on Orkambi or Kalydeco from Vertex pharmaceuticals), would be made available to people with CF in Ireland.

Cystic Fibrosis Ireland calls on the Minister for Health to ensure that children with CF who have the potential to benefit from this extension of Orkambi are given the choice of accessing this groundbreaking and innovative drug as soon as possible (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 2 copies of the F508del alteration. It is difficult to make a precise forecast of the number of additional children that will benefit from the extension of Orkambi, but we would estimate that up to 50 children in Ireland will  ultimately benefit from this new decision. 

A statement from Vertex on the Approval of Orkambi in children with CF, ages 6 - 11, with two copies of the F508del Mutation can be viewed by clicking here.

Philip Watt
Chief Executive
Cystic Fibrosis Ireland