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What is Orkambi?

Approved for use in cystic fibrosis (CF) patients ages 12 and up, Orkambi is a tablet that contains two drugs, lumacaftor and ivacaftor. It is intended for those CF patients who have two copies of the F508del mutation in the CFTR gene, which is about half of those in the United States. CF causes dangerous buildups of thick mucus in the body, causing breathing and digestive problems as well as susceptibility to diabetes and infection. The combination of medications helps the defective protein from the CFTR gene function normally. The recommended dose is once every 12 hours. Orkambi raises the risk for liver problems, and patients already suffering liver disease may not be good candidates for this medication. Side effects are unusual, but include nausea, diarrhea, gas, fatigue, and rash. Because some breathing problems, as well as upper respiratory tract infections, have been reported in users, patients new to Orkambi are monitored.

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