News & AnalysisPersonalized Medicine

New Cystic Fibrosis Drug Offers Hope, at a Price

Science  10 Feb 2012:
Vol. 335, Issue 6069, pp. 645
DOI: 10.1126/science.335.6069.645

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Summary

Last week, U.S. regulators approved a new drug for cystic fibrosis (CF). The drug, called Kalydeco and made by Vertex Pharmaceuticals, is the first to target the genetic defect discovered 23 years ago that causes a protein to malfunction in CF, resulting in a sticky buildup of mucus in the lungs and digestive tract that eventually causes fatal health problems. But the approval of Kalydeco illustrates both the promise and peril of personalized medicine, which in this case has resulted in a drug that's extremely expensive and helps only 4% of people with the disease, or 1200 patients.