The agreement between the Irish Health Service Executive and Vertex will make Orkambi available to patients 12 and older who have two copies of the F508del mutation in their CFTR gene — the gene that is defective in CF. Vertex estimates there are nearly 500 people in this category.
Kalydeco will be provided to adults with an R117H mutation and to children between 2 and 5 years old, who have a gating mutation like G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D.
If regulators expand the population eligible for the therapies, the agreement provides that the newcomers gain quick access to the treatments. This would apply to additional age groups, for example.
The national health insurance coverage is effective immediately.
“We are pleased that these additional Irish CF patients will finally join the thousands of others around the world who are already benefitting from Orkambi and Kalydeco,” Simon Bedson, a senior vice president at Vertex, said in a press release.
“We thank the leaders in Ireland for working with us toward an innovative reimbursement agreement that provides access to these important medicines and also recognizes the need for Vertex’s continued investment in the research and development of new medicines for those people with CF, many of whom are still waiting for a treatment for the underlying cause of the disease,” Bedson added.
When CF patients have two copies of the F508del mutation, cells can’t process and transport the CFTR protein correctly. This leads to little-to-no CFTR protein at the cell surface.
Kalydeco and Orkambi are the first two medicines regulators have approved for treating the underlying cause of CF. Kalydeco is a CFTR potentiator designed to help CFTR protein function normally once it reaches the cell surface. Normal functioning would help clear mucus from the airways. A build-up of mucus can lead to lung infections in CF patients.
Orkambi uses a combination of the active compound in Kalydeco, ivacaftor, and lumafactor. Ivacaftor enhances the functioning of the CFTR protein once it reaches the cell surface. Lumafactor increases the amount of mature protein at the cell surface.
In addition to Ireland, the country with the world’s highest rate of CF, Orkambi is available in the United States, Austria, Denmark, France, Germany, and Luxembourg. Kalydeco is available in 27 countries.