Monday 19th October

Kaftrio Update

The process of prescribing Kaftrio in Ireland has now officially commenced. 

The logistical task of prescribing Kaftrio is significant as many of our members are potentially eligible for this new and innovative drug therapy. 

The decision to prescribe Kaftrio is a medical one and will be decided between patients, their families and clinicians. 

Everyone who is potentially eligible for Kaftrio will be called for assessment in a specialised CF centre in the next while, but please continue to remain patient in these challenging times.

Ireland is among the first 4 countries in Europe to secure full access to Kaftrio. The reason for this is that Kaftrio is part of of the Orkambi Portfolio/Pipeline deal in 2017. 

The UK, Germany and Denmark are presently the only other European countries with access to Kaftrio.

Other countries outside Europe such as Canada, Australia and New Zealand still have no access to Kaftrio and are only recently getting access to Orkambi and Smydeco. We hope all countries will have access very soon. 

Access to Kaftrio will very likely be extended to some other patients in Ireland in months to come, including under 12 years of age - provided the clinical trials for these extensions goes well and approval is given by the EMA. 

Based on previous experience with other CFTR drugs, this broadening of access will likely be on an incremental basis, with 6-12 year olds first and then younger children

Access will also be extended in months to come as additional genotype variations are added- we understand that this is already happening. 

About 3-5% of the CF population in Ireland are unlikely to benefit from any CFTR Drug, including Kaftrio, because of their particular genotype combination. There is however continued significant investment in other therapies, including gene editing. CFI will keep you updated on these therapies. 

We encourage everyone to remain sensitive to those who don’t have access to Kaftrio at this time. 

The expected increase in lung function from Kaftrio is between 10% and 14% for those who are eligible and other potential health benefits, including significantly less exacerbations - the sudden worsening of CF leading to hospitalisation.  

As with all drug therapies each person will have a different impact from Kaftrio and as with all new drug therapies, patients should discuss any potential side effects with their clinical team. 

We know that this is an anxious time and just to assure you CFI is here to assist as much as possible.