The first drug to treat this disease is kalydeco which restores the ability of the protein to let chloride pass through cells. However, only 2,000 people with cystic fibrosis can be treated by kalydeco based on their particular gene mutations. Another problem posed by the disease is that the proteins cannot even reach the membrane. This is where Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) comes in with a new drug called lumacaftor which will aid in this particular problem. After this problem is eliminated, drugs such as kalydeco are expected to work. It is estimated that up to 28,000 patients can be helped by this combination.
Tirrell pointed out that this expanded market means bigger business for Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX). Positive data regarding this combination of drugs to treat cystic fibrosis will drive the stock, she added. Currently trading at about $67, analysts say that positive data will likely drive the stock over $100 while negative data will likely plunge it into the $40s.