I Am A Medical Professional ?


Cystic Fibrosis Ireland (CFI) has welcomed the HSE’s aim to make Orkambi available to children with CF from the beginning of February 2018, thanking the HSE and the Minister for Health, Simon Harris TD, for their “ongoing commitment to CF care in Ireland”.

This follows on from the decision of the European Medicines Agency (EMA) to extend the Cystic Fibrosis drug ‘Orkambi’ to children aged six to 11 years old, where previously it had only been available to people aged 12 years and over in Ireland and the rest of the European Union.

As part of the agreement that saw Orkambi approved for reimbursement by the Irish Government in April 2017, a ‘pipeline deal’ was made to ensure 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.

CFI has called 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, in accordance with advice from their consultant.

The organisation has stated that, while it is difficult to make a precise forecast of the number of additional children who will benefit from the extension of Orkambi, they estimate that up to 50 children in Ireland will ultimately gain from this new decision.

Orkambi treats the most common CF gene mutation in Ireland and the world, that is people who have 2 copies of the F508del alteration.