The US Food and Drug Administration has approved the first drug to treat a genetic cause of cystic fibrosis (CF), the agency announced yesterday (January 31). The drug, Vertex’s Kalydeco, was granted approval in approximately 3 months, twice as fast as normal FDA priority reviews and one of the fastest FDA approvals ever, according to a Vertex press release. It is one of the first drugs approved to correct a specific genetic defect.
CF, the most common fatal genetic disease in the Caucasian population, is caused by defective or missing CFTR proteins, cell receptors that regulate ion and water transport in and out of cells. Lack of CFTR proteins results in the buildup of a thick, sticky mucus in the lungs. Kalydeco, a twice-a-day pill, is approved for use in people with the G551D mutation in the CFTR gene. Approximately 1,200 people in the United States, or 4 percent of ...
