Orkambi Receives New Indication
September 28, 2016 - The U.S. FDA approved Orkambi® (lumacaftor/ivacaftor) with an additional indication for the treatment of Cystic Fibrosis. Orkambi, manufactured by Vertex pharmaceuticals, is a combination drug now approved to treat cystic fibrosis patients who have two mutated copies of F508del on the cystic fibrosis transmembrane conductance regulator (CFTR) gene and who are between 6 and 12 years of age (approximately 2,400 American patients). Orkambi first was approved by FDA in July 2015 for treating patients age 12 years and older who have the same genetic mutation (about 8,500 U.S. patients). It is the only drug that specifically affects F508del mutations, which are the most common ones in cystic fibrosis. Lumacaftor corrects CFTR and ivacaftor enhances it. For children between 6 and 12 years of age, the recommended dose is two tablets containing 100mg of lumacaftor and 125mg of ivacaftor once every 12 hours. To maximize its absorption, Orkambi should be taken along with foods, such as cheese, eggs and peanut butter, which contain fat. Vertex is conducting further clinical studies on Orkambi’s use for children as young as two years of age.
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