Approximately 300 paediatric CF patients with two copies of the F508del mutation are eligible to receive Vertex’s Orkambi. Credit: Vertex Pharmaceuticals.

The European Commission (EC) has approved extending the label for Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor) to treat cystic fibrosis (CF) in children aged between one and two years.

This oral treatment is indicated for paediatric patients with a cystic fibrosis transmembrane conductance regulator (CFTR) gene possessing two copies of the F508del mutation.

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Approximately 300 such patients are now eligible for the first time to receive a drug that can treat the underlying cause of the disease.

It is currently approved for use in the UK, US, Canada and Australia for treating CF patients aged one year or above.

Orkambi combines lumacaftor and ivacaftor, and aids in hydrating and eliminating mucus from the airways.

Vertex Pharmaceuticals CEO, global medicines development and medical affairs executive vice-president Carmen Bozic stated: “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.

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“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.”

In June 2023, the company signed a strategic collaboration with Lonza to set up a facility at Portsmouth, New Hampshire, US, to produce Vertex’s cell therapies for type 1 diabetes.