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Ivacaftor

Description of Ivacaftor: Ivacaftor (VX-770) is an experimental drug candidate for the treatment of cystic fibrosis,  currently under development by Vertex Pharmaceuticals and the Cystic Fibrosis Foundation.  The drug is designed to be effective in patients that have the G551D mutation in the defective protein that causes the disease. G551D, meaning that the amino acid glycine in position 551 is substituted with aspartic acid, is only found in a small percentage of cystic fibrosis patients.  The drug is currently in Phase 3 clinical trials and has showed promising results for this subset of patients. Cystic fibrosis is caused by a defect in a protein, cystic fibrosis transmembrane conductance regulator, which regulates fluid flow within cells and affects the components of sweat, digestive fluids, and mucus. VX-770 potentiates the effect of the defective protein to restore a more normal function. (source:http://en.wikipedia.org/wiki/Ivacaftor ).

KALYDECO (ivacaftor, VX-770) is Vertex's lead medicine in development for the treatment of people with cystic fibrosis. Known as a CFTR potentiator, this oral medicine in development aims to help CFTR protein function more normally once it reaches the cell surface, which is believed to help hydrate and clear mucus from the airways. Vertex retains worldwide rights to develop and commercialize KALYDECO (kuh-LYE-deh-koh). The brand name KALYDECO has been approved by the EMA and provisionally approved by the FDA, but KALYDECO has not been granted marketing authorization or approval from any regulatory authority.

About Cystic Fibrosis: CF is a life-threatening genetic disease affecting approximately 30,000 people in the United States and 70,000 people worldwide. Today, the median predicted age of survival for a person with CF is approximately 38 years. According to the 2010 Cystic Fibrosis Foundation Patient Registry Annual Data Report, approximately 4 percent of the total CF patient population in the United States have at least one copy of the G551D mutation. The most common form of CF is caused by the F508del mutation, which is present in nearly 90 percent of people with the disease.

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