uniQure Receives EMA’s CHMP Positive Opinion of Etranacogene Dezaparvovec for the Treatment of Adults with Hemophilia B
Shots:
- The EMA’s CHMP has adopted the positive opinion recommending CMA for etranacogene dezaparvovec in adult patients with hemophilia B
- The opinion was based on the P-III (HOPE-B) trial evaluating etranacogene dezaparvovec in 54 patients which showed a stable & durable increase in mean Factor IX (FIX) activity levels (with a mean FIX activity of 36.9%), 64% ABR reduction for all bleeds & 77% for all FIX-treated bleeds @6-18mos., 96% discontinued routine FIX prophylaxis with an overall 97% reduction in mean unadjusted annualized FIX consumption
- The 24mos, analysis showed a sustained & durable effect, was well-tolerated with no serious TRAEs with no correlation b/w patient AAV5 NAb levels at baseline & FIX activity
Ref: Globenewswire | Image: uniQure
Related News:- uniQure’s Hemgenix (etranacogene dezaparvovec-drlb) Receives the US FDA’s Approval for the Treatment of Hemophilia B
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