uniQure N.V. announced that its partner 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. 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.

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. 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%. Following infusion of etranacogene dezaparvovec, 96% of patients discontinued routine FIX prophylaxis.

The HOPE-B study 24 months analysis showed a sustained and durable effect of etranacogene dezaparvovec. In a clinical setting, etranacogene dezaparvovec continued to be generally well-tolerated with no serious treatment-related adverse events.