Use of Kaftrio for Younger Cystic Fibrosis Patients Confirmed

Date published: 13 January 2022

Health Minister Robin Swann has confirmed that Kaftrio will be made available to suitable patients aged six years and older in Northern Ireland for the treatment of cystic fibrosis (CF).

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Kaftrio was initially approved for use in Northern Ireland in 2020 following an agreement with its supplier Vertex Pharmaceuticals which gave access to people with CF over the age of 12 in line with a European-wide license. The recent extension means that clinically suitable children aged six and over can now benefit from Kaftrio.

The Minister said: “This decision will mean a great deal to the children with cystic fibrosis who are suitable for this treatment in Northern Ireland. This licence extension is included as part of the deal agreed with the supplier, Vertex in 2020. I am very pleased that this cutting-edge medicine will be available to more of our cystic fibrosis patient population and allow them to have a better quality of life.”

The move applies to patients who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and follows a recommendation by the European Medicines Agency (EMA).

Providing and delivering Kaftrio to patients is a complex process. This triple combination treatment (Kaftrio) combines three drugs which perform different functions – ivacaftor, tezacaftor and elexacaftor. Therefore a number of different healthcare professionals will work together to ensure patients are managed properly.

The Minister went on to say:

“When a new treatment becomes available it is fully understandable that patients and families should want immediate access to it. However, not all patients aged six to 11 with cystic fibrosis will be suitable for treatment with Kaftrio, and not everyone can be seen immediately by the multi-disciplinary teams who need to make this determination. The Health Service is working hard to make this treatment available to those who can benefit as soon as possible and I would therefore ask you to continue to be patient and let the clinical teams prioritise those patients who are in most need to be seen first.”

Notes to editors: 

  1. This product falls within the mandatory scope of the European Union-wide centralised procedure as per the Northern Ireland Protocol.
  1. The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has today also confirmed an extension to the licence for Kaftrio.
  1. This means that patients in Northern Ireland will have the same access to Kaftrio as those in GB.
  1. As of 1 January 2021, the MHRA carries out the licensing for new medicines in England, Scotland and Wales. In Northern Ireland, new medicines that fall within the scope of the European centralised procedure are licensed by the European Commission (EC) following recommendations from the European Medicines Agency (EMA).
  1. It is important to note that the issue of a marketing authorisation (license) does not provide patient access until funding decisions are made at a country level. In this case, provisions have been made across the UK to ensure patients can access this product ahead of HTA assessment. As a result, there should not be any disparity in access across the UK.
  1. Cystic fibrosis can be caused by various mutations in the gene for a protein called “cystic fibrosis transmembrane conductance regulator” (CFTR). People have two copies of this gene, one from each parent, and the disease only occurs when there is a mutation in both copies. Until now Kaftrio was used in combination with ivacaftor (Kalydeco) for treatment in patients aged 12 years and older whose cystic fibrosis is due to at least one F508del mutation in the CFRT gene.
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  1. The Executive Information Service operates an out of hours service For Media Enquiries Only between 1800hrs and 0800hrs Monday to Friday and at weekends and public holidays. The duty press officer can be contacted on 028 9037 8110.

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