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FDA approves Orkambi for children with cystic fibrosis
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Vertex Pharmaceuticals announced the FDA approval of Orkambi for treatment of cystic fibrosis in children aged 6 to 11 years who have two copies of the F508del mutation, according to a news release.
Orkambi (lumacaftor/ivacaftor) is the first and only medication approved to treat cystic fibrosis in patients with two copies of the F508del mutation, the most common mutation among patients with cystic fibrosis. Approximately 2,400 children in the United States aged 6 to 11 years have this mutation.
Previously, Orkambi was only FDA–approved for patients aged 12 years or older. With approval extended to children as young as 6 years, the total number of patients who are eligible for its use in the United States increases to approximately 11,000.
The FDA granted its approval based on results of an open-label phase 3 clinical safety study.
According to the drug’s manufacturer, Orkambi will be available for eligible children as soon as possible.
“The ability to treat children as young as [6 years] who have the most common form of the disease is an important milestone as we pursue our goal to develop medicines for all people with cystic fibrosis,” Jeffrey Chodakewitz, MD, executive vice president and chief medical officer at Vertex said in the release. “We believe it is important to treat the underlying cause of the disease as early as possible in these patients.”
In addition, Vertex intends to submit a Marketing Authorization Application variation to the European Union within the first 6 months of 2017 to extend the approval of Orkambi for younger children with two copies of F508del mutation based on the results of a phase 3 efficacy study evaluating absolute change in lung clearance index.