New Dosage Form and Expanded Indication Approved for Orkambi
August 6, 2018 – The U.S. FDA has approved a new dosage form and expanded indication for Orkambi® (lumacaftor/ivacaftor), manufactured by Vertex Pharmaceuticals, Inc. Previously approved to treat children six years of age and older, Orkambi is now indicated to treat children as young as two years of age. In addition, a new oral granules dosage form will allow the product to be more easily given to children who may have difficulty swallowing tablets.
Orkambi is indicated to treat cystic fibrosis (CF) in patients of appropriate age who are homozygous for the F508del mutation in the CFTR gene. It helps to improve the function of CFTR proteins, which are necessary to create channels that allow the body to properly transport chloride ions. Cystic fibrosis can occur when the F508del mutation interferes with the creation of these channels.
The oral tablet form of Orkambi received FDA approval in 2015. Orkambi’s oral granule form will be available in two dosage strengths, lumacaftor 100mg/ivacaftor 125mg and lumacaftor 150mg/ivacaftor 188mg, to accommodate different dosing recommendations as determined by a child’s weight and age. For administration, the granules are mixed with one teaspoon of liquid or soft food and taken by mouth. Orkambi should be taken once every 12 hours with food that contains a high amount of fat.
Vertex Pharmaceuticals has launched the new dosage form at an average wholesale price of $25,103.08 per box.