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uniQure announces FDA approval of first gene therapy for adults with hemophilia B
~ Historic approval provides a new treatment option that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy, and
About this update from Uniqure N.v.
[{"type":"text","content":"~ Historic approval provides a new treatment option that reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy, and generates elevated and sustained factor IX levels ~ ~ uniQure conducted the multi-year clinical development program and will now leverage its leading gene therapy manufacturing capabilities to supply CSL for commercialization ~ LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Nov. 22, 2022 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that its partner, global biotechnology leader CSL (ASX: CSL), has received approval from the U.S. Food and Drug Administration (FDA) for HEMGENIX® (etranacogene dezaparvovec-drlb), a one-time gene therapy for the treatment of adults 18 years of age and older living with hemophilia B. The product 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. CSL licensed the exclusive global rights to HEMGENIX from uniQure in May 2021 and is now solely responsible for the further development, registration, and commercialization of the therapy. “Today’s landmark approval represents a major milestone in the field of genomic medicine and ushers in a new treatment paradigm for patients living with hemophilia B,” said Matt Kapusta, chief executive officer of uniQure. “This is uniQure’s second internally-developed gene therapy to achieve approval and the world’s first gene therapy for hemophilia B, an historic achievement based on more than a decade of research and clinical development, as well as the tireless dedication of our employees, clinicians, patients and their families. For nearly 25 years, uniQure has been driven by a singular mission: to transform peoples’ lives by harnessing the power of gene therapy. This groundbreaking approval delivers on this promise by providing people with hemophilia B the possibility of being liberated from burdensome infusions and an ability to unlock the potential in their daily lives. We offer our sincere gratitude to the hemophilia community, without whose years of dedication and invaluable support this medical breakthrough could not have been ...