Wednesday 05th July

EMA/European Commission extends Orkambi to 1-2 year olds

Orkambi is one of the suite of four Vertex drug therapies presently available in Ireland to eligible patients on genotype and age grounds. It first became available in 2017 after an extensive CFI led advocacy campaign. 

This announcement by Vertex (view statement here) extends Orkambi, previously only available to eligible patients aged over twoyears of age, which is welcome.

Most people with CF who were formerly on Orkambi have now switched to Kaftrio, which first became available in Ireland in 2020. However there are still some patients with CF being prescribed with Orkambi.  

The four Vertex drug therapies presently available in Ireland are Kalydeco (2013), Orkambi (2017) Symkevi (2019) Kaftrio (2020 and 2023)

It is anticipated that the EMA will approve the extension of Kaftrio to two to five years olds towards the end of 2023. This follows on from such an age extension by the FDA in the United States. CFI will of course update our members when we hear further news.