Vertex Pharmaceuticals Incorporated is about to make the year’s biggest announcement in the biotechnology industry, Meg Tirrell discussed in a recent segment aired by CNBC.
According to Tirrell, Vertex Pharmaceuticals Incorporated will report data any day now about a combination of drugs that can be used to treat cystic fibrosis. Tirrell added that the report from the pharmaceutical company is one of the biggest events, according to analysts, in the biotechnology industry for this year.
Cystic fibrosis, Tirrell noted, is a rare and inherited disease that causes lung infections and other problems and can be fatal by the age of 40. It is caused by mutations to a gene known as CFTR. The report explained that when this particular gene is not normal, the proteins produced by the gene cannot help chloride escape cells. When this happens, fluid movement across a cell wall is hindered. Thick mucus is then produced in the airways and other areas.
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 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.
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