Generic molecule: ivacaftor
Company: Vertex Pharmaceuticals
Approval date: Jan. 31
The scoop: Vertex Pharmaceuticals won early approval from the FDA for its drug Kalydeco, which is the first sanctioned drug that addresses the root cause of the disease. The drug is approved for CF patients with a specific mutation. Only about 1,200 patients in the U.S. have the G551D mutation, so like most drugs with such a small population, the pill comes with a hefty price tag--$294,000 a year. But Vertex is developing the drug in combination with other compounds, with the potential of treating way more CF patients.