It is welcome news that more people with CF in Ireland will gain access to Kaftrio this month from the HSE. This follows on from the decision made by the European Medicines Agency (EMA) in April 2021 previously reported by Cystic Fibrosis Ireland and very recently approved by the HSE (see CFI website News April and March 2021)

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

With this extension, 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. This is a result of the Portfolio deal agreed between Vertex and the HSE in 2017 as part of the ‘Orkambi Pipeline/portfolio Agreement’ which is supported by CFI.

CFI estimates that around 30-40 patients who previously had no access to one of the 4 existing Vertex CFTR modulators (Kalydeco, Orkambi, Symkevi and Kafrtrio) will now have the potential to access to Kaftrio. Others who are already on an existing CFTR modulator may also gain access to Kaftrio for the first time. The final decision on access to Kaftrio or any other CFTR modulator, is of course a matter of discussion/agreement between the individual patient/their family and their CF Consultant. For general information - the HSE uses the PCRS system to reimburse expensive drug therapies https://www.hse.ie/eng/staff/pcrs/online-services/

It is important to once note again that a small percentage of patients with CF in Ireland with other rare/nonsense mutations will never be able to benefit from a CFTR modulator because they produce no CFTR protein. CFI will of course strive to continue to keep everyone informed of on-going progress in health research/clinical trials for future therapies to seek to ensure ‘no one left behind’.

CFI thanks the EMA and the HSE for this extension and thanks Vertex Pharmaceuticals for their on-going investment in CF therapy related research. As with all key drug therapies it is important that patients discuss any possible side effects with their Consultant as a normal precaution.