Cystic Fibrosis Treatment Approved for Use in Children Aged 2 to 5 Years

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Officials with the FDA have updated the label of lumacaftor/ivacaftor (Orkambi, Vertex Pharmaceuticals), approving the treatment's use in children aged 2 to 5 years old.

Officials with the FDA have updated the label of lumacaftor/ivacaftor (Orkambi, Vertex Pharmaceuticals), approving the treatment's use in children aged 2 to 5 years old; with this approval, it is now the first drug to treat the underlying cause of cystic fibrosis (CF) in this population, according to a press release.

Lumacaftor/ivacaftor is indicated for the treatment of CF in patients who have 2 copies of the F508del-CFTRmutation, the most common genetic form of the disease. The treatment has already been approved for use in patients aged 6 years and older.

This approval is based on data from a phase 3 trial involving 60 patients that showed treatment with lumacaftor/ivacaftor was safe and well tolerated for 24 weeks in this patient population. According to the data, patients demonstrated improvements in sweat chloride, a secondary endpoint, at week 24, as well as changes in key growth parameters.

Lumacaftor/ivacaftor demonstrated a safety profile similar to that in patients aged 6 years and older. The most common adverse effect associated with its use was cough and most adverse effects were mild or moderate in severity. In the trial, 4 patients experienced serious adverse events and 3 patients discontinued treatment due to treatment emergent adverse events or elevated liver function tests.

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