In very welcome news, CFI understands that Orkambi will be extended to eligible patients with CF aged one year and older from 1st December 2023. Orkambi was previously available in Ireland to eligible patients aged two years and over. 

Background: There are long-term drug therapy reimbursement agreements (also known as pipeline of portfolio agreements) in place in Ireland, Austria, Denmark and Sweden. These agreements have (in general) enabled patients in these countries to gain access to the four Vertex CFTR Modulators (Kalydeco, Symdeco, Orkambi and Kaftrio) following age and genotype extensions without an excessive delay, after approval by the EMA. 

The first such portfolio agreement (globally- for any drug therapy, we understand) was developed between Vertex and the Ireland in 2017 and has generally worked well to date. The portfolio agreement system in Ireland has since been adapted for Austria, Denmark and Sweden and we understand has also shaped agreements in some other countries outside Europe.

The 'Kaftrio 35' dispute arose (we understand) out of a disagreement between Vertex and the HSE as to whether a group of children with CF were included in Ireland's portfolio agreement or not, because of their particular genotype. As our members will know, Vertex and Ireland reached agreement on this dispute earlier this year. We thank the Department of Health/HSE for approving these continued extensions- giving hope to  people with CF in Ireland who previously could not access such life changing drugs. We further thank Vertex for their ongoing investment in research for further CF Therapies. We will continue to urge the EMA to extend the licence for CFTR Modulators, especially to those with rarer genotypes to ensure no-one who is eligible is left behind from these important therapies.