Orkambi is an oral treatment for very young children that combines both lumacaftor and ivacaftor
Vertex Pharmaceuticals has announced that the European Commission (EC) has approved the label extension of Orkambi
The therapy has been developed to treat one to <two years old with cystic fibrosis (CF) who have two copies of the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene. This is the most prevalent form of the disease.
Due to a long-standing reimbursement agreement across the UK, children in this specific age range have had access to the expanded indication for Orkambi since the Medicines and Healthcare products Regulatory Agency’s approval in March this year.
Meanwhile, reimbursement agreements in Austria, Denmark, the Republic of Ireland and Sweden – and provisions for access in healthcare systems including Germany – will ensure eligible patients have access to the therapy, following EC approval, in due course.
Orkambi is an oral treatment that combines both lumacaftor and ivacaftor. Lumacaftor is designed to increase the amount of mature protein at the cell surface by targeting the processing defect of the F508del-CFTR protein. Meanwhile, ivacaftor facilitates the ability of CFTR proteins to transport salt and water across the cell membrane.
Silvia Gartner, specialist in paediatrics and pneumonology, coordinator of the Pediatric Cystic Fibrosis Center, Barcelona, reflected: “CF symptoms and organ damage can manifest very early in life, so it is crucial to start treatment as early as possible. The approval provides us with a medicine that gives a window of opportunity to possibly delay the onset of CF for these very young eligible children.”
Carmen Bozic, chief medical officer at Vertex, was optimistic about the EC’s verdict: “This approval will offer some of the youngest children with CF the chance of improved outcomes, by treating their disease at a young age.
“With this important milestone, we move ever closer to our goal of providing medicines that treat the underlying cause of CF to all people living with the disease.”
CF remains a rare, life-shortening genetic disease impacting more than 88,000 people worldwide. The condition is a multi-organ disease that affects the lungs, liver, pancreas, sinuses, sweat glands and reproductive tract.
Vertex will continue to work with reimbursement bodies across the EU, Australia and Canada to provide access for all eligible patients.