FDA Approves Breakthrough Therapy for Cystic Fibrosis


On October 21, 2019, the U.S. Food and Drug Administration (FDA) approved a new medication to treat cystic fibrosis. The new treatment is approved for cystic fibrosis patients age 12 and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which represents roughly 90 percent of cystic fibrosis patients, or about 27,000 people in the United States. The approval of this treatment will bring hope to many cystic fibrosis patients who previously did not have any treatment options.

According to the Acting Commissioner of the FDA, Ned Sharpless, the agency used Priority Review, Fast Track, Breakthrough Therapy, and orphan drug designation to efficiently approve this new medication. Because of this medication’s importance to the cystic fibrosis community, the FDA was able to complete its review of the medication in approximately three months, greatly outpacing the original March 2020 review goal date.


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