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uniQure announces positive CHMP opinion for etranacogene dezaparvovec – gene therapy for adults with hemophilia B
If approved, etranacogene dezaparvovec would be the first licensed gene therapy in Europe for people living with hemophilia B LEXINGTON, Mass. and AMSTERDAM,
About this update from Uniqure N.v.
[{"type":"text","content":"If approved, etranacogene dezaparvovec would be the first licensed gene therapy in Europe for people living with hemophilia B\nLEXINGTON, Mass. and AMSTERDAM, Dec. 16, 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 a positive opinion recommending conditional marketing authorization (CMA) for etranacogene dezaparvovec from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). Etranacogene dezaparvovec is a one-time gene therapy for the treatment of appropriate adults with hemophilia B that reduces the rate of annual bleeds after a single infusion.1,2 uniQure conducted the multi-year clinical development program for etranacogene dezaparvovec and will leverage its leading gene therapy manufacturing capabilities to supply CSL for commercialization. “We are very pleased with this positive opinion recommending conditional marketing authorization for etranacogene dezaparvovec,” said Matt Kapusta, chief executive officer of uniQure. “The program’s multi-year clinical journey began in Europe with our first-in-human Phase I/II study, and the favorable CHMP opinion moves etranacogene dezaparvovec one step closer to the European hemophilia B community. We look forward to continuing our partnership with CSL to help bring this potentially life-changing treatment option to people with hemophilia B.” Etranacogene dezaparvovec was recently approved by the U.S. Food and Drug Administration. CSL licensed the exclusive global rights to etranacogene dezaparvovec from uniQure in May 2021, and is now solely responsible for the further development, registration, and commercialization of the therapy. uniQure is responsible for the global commercial supply of etranacogene dezaparvovec. The positive CHMP opinion is based on findings from the pivotal HOPE-B trial, the largest gene therapy trial in hemophilia B to date.3,4 These findings showed that hemophilia B patients treated with etranacogene dezaparvovec demonstrated stable and durable increases in mean Factor IX (FIX) activity levels (with a mean FIX activity of 36.9%) which led to an adjusted annualized bleed rate (ABR) reduction of 64%.1 Following infusion of et...