MarzAA, a novel variant of factor VII, specifically called Marzeptacog alfa has been reported to be very effective for treatment through prophylaxis in those with severe congenital hemophiia with inhibitors. In a study conducted as an open label phase 2 trial, MarzAA significantly decreased bleed frequency and duration of bleeding episodes by approximately 90 percent each.
The Study: In patients who suffered from spontaneous bleeding, the dose of Marzeptacog alfa was sequentially escalated until the dose amount proved effective. The dose levels were 60, 90, and 120 μg/kg. After finding an effective dose for said patient and after 50 days with no spontaneous bleeding, the patients were evaluated in a 30-day follow-up period. The conclusion was reduction in annualized bleeding rate for Marzeptacog alfa. The secondary endpoints were tolerability, safety, and antidrug antibody formation. 11 male participants, 10 having hemophilia A and 1 having hemophilia B, were treated with MarzAA. The median age of the 11 male participants was 31 years old with a range of 18-47 years old. The annualized bleed rate of hte participating participants was 19.8.
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