As CFI members will be aware, in December 2019 Vertex and the HSE confirmed that they had successfully negotiated an expanded agreement for the use of the new and innovative CFTR drug therapy ‘Trikafta’* in the Republic of Ireland, once it has been licensed by the European Medicines Agency (EMA). Trikafta will likely supersede Orkambi and Symdeco/Symkevi for many patients, once it becomes available in Ireland (protocols will be developed to guide specialised CF centres following EMA approval of Trikafta).

The 2019 expanded agreement originated from the 2017 pipeline agreement that secured Orkambi and Symdeco/Symkevi and subsequent age and other expansions for existing CFTR drugs. While a few commentators in Ireland were cynical towards the Orkambi pipeline deal (sometimes because they did not fully understand it), it has been widely hailed by many Pharma analysts worldwide as being a new and innovative approach to ensure access to vital and proven drug therapies in double quick time. Denmark has adopted a similar approach.

Ludovic Fenaux, Senior VP of Vertex recently stated in respect of Ireland
'With this expanded agreement, even more patients in the Republic of Ireland will be among the first in Europe to benefit from the triple combination treatment once the medicine is licensed.'

The precise date when Trikafta will be approved by the EMA is still unknown but CFI predicts that the decision is now more likely in the autumn/winter of 2020. There had been some initial hope that Trikafta would have been fast-tracked by the EMA which would have made it available in Ireland in June/July 2020, but this is now unlikely, but we believe for solid reasons.

CFI understands the likely reason for non-fast tracking of Trikafta is because it would be important that the EMA license for Trikafta is as broad as possible, i.e. covering as many altered gene combinations as possible.

In short, if it had been fast tracked by the EMA, some people who should have been eligible for Trikafta in Ireland would have experienced a delay of many months, perhaps even longer. It makes sense that as many eligible patients as possible benefit from Trikafta, so non-fast tracking is, on balance, the most sensible approach, particularly if it only means a difference of 3 or 4 months.

For those PWCF who will not be able to benefit from Trikafta or another CFTR drug CFI again promises ‘no one left behind’ and we will continue to support and strive for other new and innovative therapies, working with clinicians and researchers. Look out for Dr Patrick Harrison who will be speaking at our annual conference on the potential of gene editing (available by live podcast also).

* Trikafta is the triple combination regimen of elexacaftor, tezacaftor and ivacaftor -  it will likely be rebranded (shortly) under a different name in Europe.