UniQure and CSL Published P-III Study (HOPE-B) Results of Hemgenix (etranacogene dezaparvovec-drlb) for Hemophilia in NEJM
Shots:
- The P-III study evaluating Hemgenix (2x1013 gc/kg, IV) in 54 adult patients showed that Hemgenix was superior to routine factor IX prophylaxis in ABR, improvement in factor IX activity, factor IX therapy consumption, factor IX infusion rate, spontaneous & joint bleeding ABR, increased factor IX activity from 3wk. & maintained ~18mos. with no serious TRAEs
- Reduction of 71% & 78% in ABR of spontaneous & all joint bleeding episodes from the lead-in period to post-treatment, 96.3% discontinued factor IX prophylaxis from 21 Day through 18mos.
- Annualized factor IX infusions also decreased from 72.5 infusions/participant during the lead-in period to 2.5 infusions post-treatment, Hemgenix was approved in the US in Nov 2022 & in the EU in Feb 2023 for adults
Ref: PRNewswire | Image: UniQure
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