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The Food and Drug Administration announced on Monday (October 21)—months ahead of its expected decision date—that it has approved a new treatment for cystic fibrosis. Trikafta, made by Vertex Pharmaceuticals, is, as its name suggests, a combination of three drugs. It works for 90 percent of patients with cystic fibrosis, and will be priced at $311,000 per year.
“Now, I know that my son’s lungs will function as well as mine. That’s just unbelievable,” Margot Cleveland, whose 10-year-old son has cystic fibrosis, tells STAT of the approval.
Cystic fibrosis is a rare genetic disorder that affects about 30,000 people in the United States. It can be caused by a variety of mutations in the gene CFTR, which codes for a protein involved in moving salt in and out of cells. People with the disease produce unusually sticky mucus that plugs up the lungs and other organs. Trikafta ...
