Cystic Fibrosis Ireland understands that Kaftrio will begin to become available in Ireland from October 2020 onwards, as we previously predicted.
Those who are potentially eligible to benefit from Kaftrio will be contacted by their CF team for an assessment before a prescribing decision is made. This is a strictly medical issue and CFI has, of course, no role in determining who will be prescribed Kaftrio.
In some cases, the decision may be complex because of the existing complexities of CF and treatment. The decision of access to Kaftrio is one that can only be taken in discussion between the person with CF/their family and their CF consultant.
It should be noted that not everyone who is potentially eligible for Kaftrio will receive Kaftrio in the next few weeks. For example, Kaftrio is presently only licensed for people with CF aged 12 years and over. In future months, it is very possible that Kaftrio will be extended to younger age groups (once the EMA has approved such extensions). This can only be based on evidence from clinical trials on efficacy and safety and other research.
Because of the high numbers of people in Ireland who are potentially eligible for Kaftrio, the process of assessment is likely to take some time, so we ask for continued patience and understanding at this time.
CFI understands that there is very little chance of CFTR drugs, including Kaftrio, being delayed because of supply problems related to BREXIT or COVID-19.
About Kaftrio
Kaftrio is a medicine used to treat eligible patients aged 12 years and above who have cystic fibrosis.
Kaftrio is approved in Europe for the treatment of cystic fibrosis (CF) in patients ages 12 years and older with one F508del mutation and one minimal function mutation or two F508del mutations in the CFTR gene.
Kaftrio is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface
Cystic fibrosis can be caused by various mutations (changes) in the gene for a protein called ‘cystic fibrosis transmembrane conductance regulator’ (CFTR). People have 2 copies of this gene, one inherited from each parent and the disease only occurs when there is a mutation in both copies.
Kaftrio is used in patients whose cystic fibrosis is due to the F508del mutation inherited from one or both parents. If they have inherited this mutation from only one parent, they should also have another mutation called a ‘minimal function mutation’ from the other parent.