The first drug that targets an underlying cause of cystic fibrosis rather than the symptoms -- essentially allowing some sufferers to live a nearly normal life -- has been approved by the Food and Drug Administration.
The drug Kalydeco (also known by its generic name, ivacaftor) helps a defective protein that acts as a chloride-pump in cell membranes to function properly, restoring the proper flow of water in the body. As a result, patients' mucus-filled lungs begin to clear, making them less prone to infection and perhaps allowing them to live far beyond the age of 37 -- the current average life expectancy.
“Kalydeco is an excellent example of the promise of personalized medicine -- targeted drugs that treat patients with a specific genetic makeup,” FDA Commissioner Dr. Margaret A. Hamburg said in a news release. The FDA fast-tracked Kalydeco and approved it in three months, given the drug's early signs of promise.
For now, the drug helps only the 4% to 5% of cystic fibrosis patients with a particular genetic mutation. But drug maker Vertex Pharmaceuticals aims to develop other drugs that, used in combination with ivacaftor, may help many more patients as well.