X-ray of the lungs of a cystic fibrosis patient. Credit: Science Photo Library.

The US Food and Drug Administration (FDA) has granted approval to Vertex Pharmaceuticals for the use of its Orkambi (lumacaftor/ivacaftor) medicine to treat cystic fibrosis in children between two and five years old.

Indicated for kids with two F508del-CFTR mutation copies, Orkambi is said to be the first FDA-approved drug for targeting the underlying cause of the disease in this population.

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Orkambi is comprised of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the F508del CFTR protein, and ivacaftor that improves the CFTR protein function upon reaching the cell surface.

Vertex Pharmaceuticals executive vice-president and chief medical officer Reshma Kewalramani said: “For the first time, children ages two through five who have the most common form of CF have a treatment for the underlying cause of their disease.

“We believe it is important to treat the underlying cause of the disease as early as possible and this approval is another significant milestone in our journey to bring effective medicines to all people living with CF.”

“This approval is another significant milestone in our journey to bring effective medicines to all people living with CF.”

The FDA decision comes after review of data from 60 patients who participated in a Phase III open-label safety study, which showed that Orkambi is safe and well-tolerated for 24 weeks. This safety profile is said to be similar to that observed in patients of six years and above age.

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While most common adverse events were mild or moderate in severity, four subjects experienced serious events and three had to discontinue the therapy because of treatment emergent adverse events or increased liver function tests.

Previously, Orkambi obtained the FDA approval for cystic fibrosis in patients aged six and older with two copies of the F508del-CFTR mutation.

Vertex has also submitted a regulatory filing to the European Medicines Agency (EMA) for the use of the drug in patients aged two through five years.

Orkambi has already been approved by the EMA for children over the age of six. However, it is not currently available through the UK’s National Health Service (NHS) because Vertex and NHS England are unable to agree on an acceptable price for the drug.