World's most expensive drug: USFDA nod to HEMGENIX worth USD 3.5 million a dose

Global biotechnology leader CSL has announced that the U.S. Food and Drug Administration (USFDA) has approved HEMGENIX (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy for appropriate adults with hemophilia B. HEMGENIX is approved for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes. In the ongoing clinical trial, HEMGENIX reduced the rate of annual bleeds and 94 percent of patients discontinued factor IX prophylaxis and remained prophylaxis-free.

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FIRST: CSL Bags USFDA Approval For HEMGENIX To Treat Hemophilia B