Landmark decision on ‘Trikafta’ (now to be known as ‘Kaftrio’ in Europe)
A further important landmark in the approval process for the triple combination CFTR drug therapy now known as ‘Kaftrio’ (formerly known as Trikafta) has been reached today (26 June 2020). However, there is still some important further work needed before this drug therapy finally becomes available in Ireland, still likely to be in Autumn 2020, if all goes well.
CFI provides the following information in the form of Frequently Asked Questions (FAQ’s) from the information we know to date. We will update you as further information becomes available.
Does the new information published today relate to Trikafta?
Yes. ‘Trikafta’, or the ‘Triple Combination Drug’ from Vertex, will now be known as ‘Kaftrio’ in Europe (including Ireland).
It is often normal practice that new drugs have a different name in Europe compared to the United States of America. The drug therapy will continue to be known as ‘Trikafta’ in the USA.
What is the good news announced today by the CHMP of the EMA?
Today marks a very important stage in the approval process for Kaftrio by the European Medicines Agency (EMA). The Committee for Medicinal Products for Human Use (CHMP) - which is the most important committee within the European Medicines Agency (EMA) - has recommended approval of the triple combination drug therapy 'Kaftrio'.
Does this mean that Kaftrio is now available in Ireland and the rest of the EU?
Not yet, but likely very soon. Kaftrio still has to be given final approval by the European Commission. There is normally a gap of on average 55 days between a CHMP recommendation and the final decision on a drug’s approval by the European Commission.
In most other countries in the EU, following approval by the European Commission, Kaftrio would then then have to be considered for reimbursement by the national medicines agency in each country (for example, the NCPE in Ireland).
Thanks to the innovative ‘Portfolio Agreement’ between the HSE and Vertex (signed in 2017) which provided for Orkambi and the extension of existing and forthcoming CFTR drugs (which was strongly supported by CFI) as soon as the European Commission approves Kaftrio, it will start to become available in Ireland without having to go through the HTA assessment and related HSE processes.
There will then be a logistics phase when the HSE, Vertex and CF specialised centres prepare for Kaftrio in Ireland. This will involve a considerable amount of work, not least because of the potential numbers of people with CF involved.
Will everybody with CF in Ireland benefit from Kaftrio?
No, but many will have the potential to benefit - if not at time of approval - then in forthcoming months.
It is estimated that as much as 90% of the CF population worldwide have the potential to benefit from Kaftrio/Trikafta, but we have to wait and see the final percentage that may benefit in Ireland. Like other CFTR drugs, Kaftrio will likely be introduced into Ireland incrementally. It is important to note, for example, that the present recommendation from the CHMP is for people with CF who are 12 years and over.
We will also have to wait and see the full extent of the license of Kaftrio for Europe/Ireland as the CHMP judgement is different from that of the Food and Drug Administration (FDA) in the United States of America. However, as with other CFTR drugs such as Kalydeco, Orkambi and Symkevi, there will also likely be extensions of Kaftrio in the months following first approval.
It is known already (see CFI website news) that not all people with CF in Ireland can benefit from a CFTR drug (such as Kaftrio) because of their genotype combination or other reasons. There is continuing high investment in other possible CF therapies including for example, gene therapy and gene editing. CFI continues to urge 'no one left behind'.
Will there be side-effects from Kaftrio?
Yes, almost all drug therapies have potential side effects and it will be important when the time comes to read the information about Kaftrio very carefully and talk over any concerns that you may have with your consultant if you are eligible for this drug therapy.
Next Steps
The likely next steps in Ireland are to wait until the European Commission makes a final judgement on Kaftrio and to look-out for any statements from the Minister for Health. At the start of the process, CFI predicted that this drug therapy could be available in Ireland in the Autumn of 2020- this still looks like the likely timescale, but it could be subject to change, for example we don't know the full impact of delays arising from COVID-19 that may alter the logistics phase.
It’s another important day for CF care in Ireland but patience is still needed. If all goes to plan, people with CF in Ireland, Denmark and Germany will have first access to Kaftrio in Europe.
CFI wishes to thank all who have been involved in getting us to this stage, including in particular people with CF and their families, the Board and Staff of CFI, the HSE, the NCPE and the outgoing and incoming Government and indeed all political parties, our valued Hospitals, Consultants and their MDT's and of course Vertex Pharmaceuticals for developing these wonderful new CFTR drugs .