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uniQure Receives EMA’s CHMP Positive Opinion of Etranacogene Dezaparvovec for the Treatment of Adults with Hemophilia B

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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

Click here to­ read the full press release 

Senior Editor

Neha is a Senior Editor at PharmaShots. She is passionate and very enthusiastic about recent updates and developments in the life sciences and pharma industry. She covers Biopharma, MedTech, and Digital health segments along with different reports at PharmaShots. She can be contacted at connect@pharmashots.com.

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