uniQure’s Hemgenix (etranacogene dezaparvovec-drlb) Receives the US FDA’s Approval for the Treatment of Hemophilia B


The US FDA has approved Hemgenix (etranacogene dezaparvovec-drlb) in adults aged ≥18yrs. with hemophilia B
The approval was based on the P-III (HOPE-B) trial evaluating Hemgenix (2×1013 gc/kg, IV) in 54 patients which showed a reduction in the rate of annual bleeds & 94% of patients discontinued factor IX prophylaxis and remained prophylaxis-free with mean factor IX activity of 39% @6mos. and 36.7% @24mos. post-infusion, 54% reduction in ABR bleeds over 6mos. on factor IX prophylactic replacement therapy
Under the terms of the agreement, CSL gets an exclusive right globally to Hemgenix in May 2021 & responsible for the development, registration & commercialization of the therapy

Ref: Globenewswire | Image: uniQure