The European Commission has granted approval for the label extension of Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor) for the treatment of children with cystic fibrosis (CF) ages 1 to under 2 years old who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common form of the disease.

Orkambi was previously approved to treat cystic fibrosis in patients aged 2 years and above. Now, nearly 300 children with cystic fibrosis and two copies of the F508del mutation are eligible for the first time for a medicine that can treat the underlying cause of their disease, according to a release from Vertex. 

“This approval will offer some of the youngest children with cystic fibrosis the chance of improved outcomes by treating their disease at a young age,” says Carmen Bozic, MD, executive vice president, global medicines development and medical affairs, and chief medical officer at Vertex, in a release. “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.”

Orkambi has also been approved by regulatory authorities in the United States, Great Britain, Australia, and Canada, for people with CF and two copies of the F508del mutation in the CFTR gene, ages 1 and above.

“CF symptoms and organ damage can manifest very early in life, so it is crucial to start treatment as early as possible,” says Silvia Gartner, MD, specialist in pediatrics and pneumonology, coordinator of the Pediatric Cystic Fibrosis Center, Barcelona, in a release. “Today’s 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.”

As a result of long-term reimbursement agreements in Austria, Denmark, the Republic of Ireland, and Sweden, and provisions for access in health care systems such as Germany, eligible patients in these countries will have access to the expanded indication for Orkambi shortly following regulatory approval by the European Commission. 

As a result of the long-term reimbursement agreement in the UK, children ages 1 to under 2 years old in the UK have had access to this expanded indication for Orkambi since Medicines and Healthcare products Regulatory Agency approval in March 2023. Vertex will continue to work with reimbursement bodies across the European Union, Australia, and Canada to ensure access for all eligible patients, according to a release from the company.