CFI welcomes today's expected decision by the European Commission to widen the eligibility of the innovative and ground breaking CF drug Kaftrio to more patients in Ireland.  

The recommendation from the Committee for Medicinal Products for Human Use (CHMP) of the EMA, previously reported by CFI on 26th March 2021, has now been formally approved by the European Commission.  

Thanks to the portfolio/pipeline agreement that was supported by CFI in 2017 as part of our Orkambi campaign, the additional eligible patients with CF aged 12 years and over will be able to gain access to Kaftrio in weeks to come, provided too of course they consent and approval is given by their clinical teams.  

People with CF, aged 12 and over, who have one copy of the F508del mutation, regardless of their other mutation type will be eligible for Kaftrio. 

With this extension of the indication, CF patients 12 years and older who are heterozygous for the F508del-CFTR mutation and a residual function (F/RF) or gating mutation (F/G) are eligible for the triple combination therapy for the first time, and the majority of people ages 12 years and older with CF in Europe are now eligible for Kaftrio.  

This will mean, for example that some patients currently on Kalydeco, may decide to switch to Kaftrio if approved by their clinical teams. Others may wish to continue with Kalydeco.  

Please note and be sensitive to the fact that while this is further good news, some people with CF will not benefit from this decision or indeed any further widening of access to Kaftrio or other CFTR modulators in months to come and further research into other types of CF therapies is still very much needed - 'no one left behind’.  

The press release from Vertex issued today, 28th April, is included below for information.