Though there are other treatments available to help manage symptoms, cystic fibrosis does not currently have a cure. That’s part of what makes Orkambi (ivacaftor/lumacaftor) exciting—this new medication targets the two defective genes that are the underlying cause of the disease.
Now, after receiving FDA approval on July 2, 2015, Orkambi is available in pharmacies.
What is cystic fibrosis?
Cystic fibrosis (CF) is an inherited and life-threatening disease that affects the respiratory and digestive systems. It mainly affects the lungs, but can also involve the pancreas, liver, intestine, sinuses, and sex organs.
Symptoms can include coughing with thick mucus or blood, lung infections, and diarrhea, though these vary from person to person.
What causes cystic fibrosis?
Cystic fibrosis is caused by a mutation of the CFTR gene.
A child will develop cystic fibrosis if they inherit two copies of a mutated gene, one from each parent (neither of whom is likely to have the disease themselves).
How does the gene mutation affect someone with CF?
The mutation causes interference with the transportation of water and chloride in the body. That interference leads to the buildup of thick, sticky mucus that can damage many of the body’s important organs.
How is Orkambi taken?
What are the most common side effects associated with Orkambi?
Some common side effects include shortness of breath, upper respiratory symptoms, nausea, diarrhea, rash, and increased menstrual bleeding.
Will Orkambi be available at my pharmacy?
No. Orkambi will be available exclusively at specialty pharmacies—you won’t be able to go to your regular retail pharmacy to fill a prescription. You can see a list of the specialty pharmacies that will offer Orkambi here and here.
More worrisome? Orkambi comes with a very high price tag, at an estimated $259,000 per year. Vertex, Orkambi’s manufacturer, does offer support for getting coverage, and may offer assistance for patients in need. See their website for more information.
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