February 1, 2012- The Food and Drug Administration has given its approval for the first treatment to combat the underlying cause of cystic fibrosis. The drug attacks the genetic mutation that affects a small number of patients that suffer from the breathing disorder that can be fatal. Vertex Pharmaceuticals makes the drug, Kalydeco. The treatment will be one of the most expensive treatments offered anywhere with a cost of over $294,000 per year.
CF causes the sufferers body to produce sticky and unusually thick mucus that cause the lungs to be clogged, and by obstructing the pancreas stops enzymes from breaking down and absorbing food. Patients get infections easily and progressively their ability to breathe gets worse.
Before Tuesday’s FDA approval, the 70,000 cystic fibrosis patients worldwide could only take drugs that attacked the disease’s symptoms. These antibiotics for the most part treated infections, while patients normally spent up to a couple of hours daily wearing vests that would vibrate to dislodge the build-up of mucus.
The new treatment can be taken twice each day and is approved to treat those with the gene mutation that causes CF in about 4% of all the cystic fibrosis sufferers. That is about 1,200 of the estimated 30,000 that have CF in the U.S.