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World’s Most Expensive Drug Approved to Treat Hemophilia at $3.5 Million a Dose

  • CSL Behring’s hemophilia B treatment Hemgenix approved by FDA
  • Hemgenix is one-time gene therapy administered by IV infusion
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US regulators approved CSL Behring’s hemophilia B gene therapy, a one-off infusion that frees patients from regular treatments but costs $3.5 million a dose, making it the most expensive medicine in the world. 

CSL Behring’s Hemgenix, administered just once, cut the number of bleeding events expected over the course of a year by 54%, a key study of the therapy found. It also freed 94% of patients from time-consuming and costly infusions of Factor IX, which is currently used to control the potentially deadly condition.