Vertex has won a green light in Europe for a licence extension that makes it the first approved medicine to treat the underlying cause of CF in children.
The US biotech’s Orkambi is now the first medicine approved in the region to treat 6-11 year olds who carry two copies of the f580del mutation.
Orkambi, a twice-daily drug, is a combination of lumacaftor and ivacaftor, designed to enhance the function of the CFTR protein once it reaches the cell surface.
Patients with CF carry little-to-no CFTR protein at the cell surface due to inheriting two defective CFTR genes and according to Vertex, there are approximately 3,400 6-11 year olds who carry two copies of the mutation.
Simon Bedson, senior vice president and international general manager at Vertex was keen to highlight the market access work the company has undertaken to help it reach patients.
He said: “The innovative long-term agreements we have reached in countries like Ireland will enable eligible children to have rapid access to Orkambi.
“Where these agreements are not in place, Vertex is committed to working with local authorities so those who could benefit from this medicine are able to do so as quickly as possible.”
Orkambi’s new approval was based on data from two phase III studies showing a statistically significant improvement in lung function among children treated with the medication compared to placebo.